U.S. Orphan Drug Market Size, Competitors, Trends & Forecast

Report Findings & Highlights:

US Orphan Drugs Market Opportunity: >US$ 200 Billion by 2031
Insight on Orphan Drugs in Clinical Trials: >800 Orphan Drugs
Clinical Trials Insight by Company, Indication, Phase & Priority Status
Clinical Insight on Marketed Orphan Drugs: >500 Orphan Drugs
Pricing & Dosage Insight: >400 Marketed Orphan Drugs
FDA Designated Orphan Drug Sales Insight (2021-2025): 150 Orphan Drugs
Insight on Orphan Designation by Indication, Company, Trial Phase, Marketed Drugs: >1400 Orphan Drugs

FDA Designated Orphan Drug Designation & Why This Report?

The FDA designated orphan drug market has established itself as a key platform upon which pharmaceutical innovation is built. Orphan designation is no longer utilized as a last resort approach in research and development but rather as a first mover strategy for differentiated assets with a clear regulatory and development roadmap.

This report provides a strategic view on US orphan drug market which is projected to continue its pattern of growth over the coming years. The consolidated information provided here includes a series of critical insights into development pipelines, partnerships, technology platforms, geographical footprint, clinical strategies, and competitive position. As orphan designated programs are being advanced through the drug development process, this report allows for an informed decision-making approach based on investment, partnership, and portfolio strategy.

Significantly, the report also points out the larger ecosystem that enables the development of the orphan drug sector, including the contribution of public organizations, academic research institutions, and technology licensing organizations that have traditionally facilitated early research in the treatment of rare diseases.

FDA Designated Orphan Drug Clinical Trials Insights Covered in the Report

Clinical trial intelligence is vital for understanding the orphan drug market because clinical development of these drugs is characterized by small patient populations, unique trial endpoints, and expedited regulatory tracks. The report provides analysis of clinical trial data by indications, clinical development phases, sponsors, and strategies, enabling readers to gain insights into clinical development of orphan drugs.

The report also points out the key contribution that institutional sponsors and academic collaborators make to early and mid-stage trials of orphan drugs. Academic centers and research hospitals frequently serve as initiators, patient identification centers, and partners in translational science, especially within ultra-rare diseases. These centers may also often assist new biotech companies to define proof of concept or de-risked mechanisms of action before entering a clinical development pipeline with larger sponsors.

The report shows that some key cases were used to illustrate how companies use orphan designation to deliver targeted clinical strategies in rare oncology and genetic disorders, including development programs with multiple indications based on common underlying biology or molecular mechanisms. While it shows some trends in trial design efficiency and collaboration between sponsors, it does not reveal operational details of such programs.

Leading Companies Active in R&D on FDA Designated Orphan Drugs

The orphan drug market space is primarily supported by high participation levels of both large pharmaceutical companies and smaller biotechnology organizations. Established industry leaders continue to drive growth and increase orphan product development within oncology, hematology, immunology, and rare genetic disorders.

Companies like Roche, Bristol Myers Squibb, Novartis, Sanofi, Gilead Sciences, Takeda, and Johnson & Johnson have diversified orphan drug pipelines with ongoing investment in internal R&D, as well as external collaborations and strategic acquisitions. This indicates their long-term commitment and recognition of the value of orphan drug investment in their R&D and overall business strategy.

Report Indicating the Future Development of FDA Designated Orphan Drugs

The prospects for the development of future orphan drugs are expected to be influenced by improved precision in patient targeting, increasing modality, and economies of scale using platform based strategies. The development process for orphan drugs has benefited from enhanced molecular diagnostics with more precision-focused approaches, particularly for cancer therapies and genetic disorders, as well as novel biologics, gene therapies, and RNAs for wider rare disease modality development.

in this context, the report also analyses the expansion of developed therapies to further rare indications and hence reinforce long-term value creation. An example of this approach is the further development of Bizengri into molecularly defined cancers like NRG1 fusion-positive cholangiocarcinoma.

Thus, the report recognizes the US orphan drug market as a long term opportunity, underpinned by a well-regulated environment, scientific innovation, and a more interconnected drug development process.

1. Research Methodology2. FDA Orphan Designation Criteria & Market Exclusivity

3. US Orphan Designated Drugs Market Insight

3.1 Current Market Overview
3.2 Future Market Opportunity Assessment

4. US Orphan Designated Drugs Market Trends by Indication

4.1 Cancer
4.2 Neurological Disorders
4.3 Cardiovascular Disorders
4.4 Ophthalmic Diseases
4.5 Rare Genetic Disorders & Metabolic Disorders
4.6 Autoimmune & Inflammatory Diseases

5. US Orphan Designated Drugs Reimbursement Scenario

5.1 Medicare
5.2 Medicaid
5.3 Private Insurers
5.4 Pharmaceutical Companies

6. US Orphan Designated Drugs Clinical Trials by Company, Indication & Phase

6.1 Research
6.2 Preclinical
6.3 Phase 0
6.4 Phase-I
6.5 Phase-I/II
6.6 Phase-II
6.7 Phase-II/III
6.8 Phase-III
6.9 Preregistration
6.10 Registered

7. Marketed Orphan Designated Drugs Clinical Insight by Company & Indication

8. US Orphan Designated Drugs Dosage, Price & Treatment Cost Insight

8.1 Lynparza
8.2 Nplate
8.3 Tafinlar
8.4 Halaven
8.5 Jadenu
8.6 Xpovio
This Chapter Gives Insight on Dosage, Price & Treatment Cost of More Than 400 FDA Designated Orphan Drugs Available in US Market
8.424 Qfitlia
8.425 Zevaskyn

9. US Orphan Designated Drugs Sales Insight (2021-2025)

9.1 Amvuttra
9.2 Krazati
9.3 Tecvayli
9.4 Imjudo
9.5 Rezurock
9.6 Elrexfio
This Chapter Gives Annual & Quaterly SalesInsight on 151 FDA Designated Orphan Drugs Available in US Market
9.149 Keytruda
9.150 Pemazyre
9.151 Camzyos

10. Competitive Landscape

10.1 AbbVie
10.2 Alnylam Pharmaceuticals
10.3 Amgen
10.4 Amicus Therapeutics
10.5 Array BioPharma
10.6 Astellas Pharma
10.7 AstraZeneca
10.8 Bayer HealthCare
10.9 BioMarin Pharmaceutical
10.10 Bioverativ
10.11 Boehringer Ingelheim
10.12 Bristol-Myers Squibb
10.13 Chiesi
10.14 Chugai Pharmaceutical
10.15 Collaborations Pharmaceuticals
10.16 CSL
10.17 Daiichi Sankyo Company
10.18 Eisai Co Ltd
10.19 Eli Lilly & Company
10.20 Emergent BioSolutions
10.21 Ferring Pharmaceuticals
10.22 Flavocure Biotech
10.23 Genentech
10.24 Genmab
10.25 Gilead Sciences
10.26 GSK
10.27 Hanmi Pharmaceutical
10.28 Incyte Corporation
10.29 Ionis Pharmaceuticals
10.30 Ipsen
10.31 Jazz Pharmaceuticals
10.32 Johnson & Johnson
10.33 Kamada
10.34 Krystal Biotech
10.35 Kyowa Kirin
10.36 Leadiant Biosciences
10.37 Ligand Pharmaceuticals
10.38 Lundbeck
10.39 MeiraGTx
10.40 Merck
10.41 Moderna Therapeutics
10.42 Novartis
10.43 Novo Nordisk
10.44 Omeros Corporation
10.45 Onyx Pharmaceuticals (Amgen)
10.46 Otsuka Pharmaceutical
10.47 Pfizer
10.48 PTC Therapeutics
10.49 Recordati
10.50 Regeneron Pharmaceuticals
10.51 Roche
10.52 Sanofi
10.53 Servier
10.54 Shionogi
10.55 Sumitomo Pharma
10.56 Sutro Biopharma
10.57 Swedish Orphan Biovitrum
10.58 Takeda
10.59 Teva Pharmaceutical Industries
10.60 UCB
10.61 UniQure
10.62 Vertex Pharmaceuticals
10.63 XOMA

List of Figures

Figure 3-1: US - FDA Designated Orphan Drugs Approval, 2020-2025
Figure 3-2: US - Orphan Designated Drugs Market Size (US$ Billion), 2024-2031
Figure 6-1: Global - Number of Orphan Drugs Clinical Trials by Phase, 2026-2031
Figure 8-1: US - Lynparza Approvals by Indication
Figure 8-2: US - Lynparza Orphan Drug Designation Years by Indication
Figure 8-3: US - Cost of 60 & 120 Tablets Supply of 100 mg & 150 mg Lynparza (US$)
Figure 8-4: US - Monthly Cost of Treatment with 100 mg & 150 mg Lynparza Tablets (US$), February’2026
Figure 8-5: US - Cost of 125 mcg, 250 mcg & 500 mcg supply Packs of Nplate (US$), February’2026
Figure 8-6: US - Tafinlar Approvals by Indication
Figure 8-7: US - Tafinlar Orphan Drug Designation Years
Figure 8-8: US - Cost Per Unit & Supply of 120 Capsules of 50mg & 75mg Tafinlar (US$), February’2026
Figure 8-9: US - Cost Per Unit & Supply of 210 Tablet 10mg Tafinlar (US$), February’2026
Figure 8-10: US - Monthly Cost of Tafinlar Treatment by Weight (US$), February’2026
Figure 8-11: US - Halaven Approvals by Indication
Figure 8-12: US - Monthly Cost of Treatment with Havalen Injection (US$), February’2026
Figure 8-13: US - Jadenu Orphan Drug Designation Years by Indication
Figure 8-14: US - Cost Per Unit & Supply of 30 Tablets of 90mg, 180mg & 360mg Jadenu (US$), February’2026
Figure 8-15: US - Monthly Cost of Treatment with Jadenu (US$), February’2026
Figure 8-16: US - Xpovio Orphan Drug Designation & Approval Year by Indication
Figure 8-17: US - Cost Per Unit & Supply of 40mg Xpovio Tablets (US$), February’2026
Figure 8-18: US - Cost Per Unit & Supply of 10mg, 20mg, 50mg, 60mg, 80mg Xpovio Tablets (US$), February’2026
Figure 8-19: US - Gavreto Orphan Drug Designation & Approval Year by Indication
Figure 8-20: US - Cost Per Unit & Supply of 100mg Gavreto Tablets (US$), February’2026
Figure 8-21: US - Monthly Cost of Treatment with Gavreto Capsule (US$), February’2026
Figure 8-22: US - Cost Per Unit & Supply of 5mg & 10mg Ocaliva Tablets (US$), February’2026
Figure 8-23: US - INOmax Orphan Drug Designation & Approval Year
Figure 8-24: US - Truseltiq Orphan Drug Designation & Approval Year
Figure 8-25: US - Cost Per Unit & Supply of Different Truseltiq Doses (US$), February’2026
Figure 8-26: US - Lumakras Orphan Drug Designation & Approval Year by Indication
Figure 8-27: US - Cost Per Unit & Supply of 120mg & 320mg Lumakras Doses (US$), February’2026
Figure 8-28: US - Monjuvi Orphan Drug Designation & Approval Year by Indication
Figure 8-29: US - Onpattro Orphan Drug Designation & Approval Year
Figure 8-30: US - Cost Per Unit & Supply of 5mL Vial of Onpattro (US$), February’2026
Figure 8-31: US - Cost Per Unit & Supply of 10 mg/mL Opdivo (US$), February’2026
Figure 8-32: US - Opdualag Orphan Drug Designation & Approval Year
Figure 8-33: US - Cost Per Unit & Supply of Opdualag Solution (US$), February’2026
Figure 8-34: US - Orladeyo Orphan Drug Designation & Approval Year
Figure 8-35: US - Cost Per Unit & Supply of Orladeyo Capsule (US$), February’2026
Figure 8-36: US - Rubraca Orphan Drug Designation & Approval Year
Figure 8-37: US - Cost Per Unit & Supply of Rubraca Tablet (US$), February’2026
Figure 8-38: US - US - Monthly Cost of Treatment with Rubraca Tablet (US$), February’2026
Figure 8-39: US - Livmarli Orphan Drug Designation & Approval Year by Indication
Figure 8-40: US - Cost Per Unit & Supply of 9.5 mg/mL Livmarli Oral Liquid (US$), February’2026
Figure 8-41: US - Cost Per Unit & Supply of 10mg & 15mg Livmarli Oral Tablets (US$), February’2026
Figure 8-42: US - Bendeka Orphan Drug Designation & Approval Year by Indication
Figure 8-43: US - Cost Per Unit & Supply of Bendeka Solution (US$), February’2026
Figure 8-44: US - Ryanodex Orphan Drug Designation & Approval Year
Figure 8-45: US - Firdapse Orphan Drug Designation & Approval Year
Figure 8-46: US - Cost Per Unit & Supply of 10mg Firdapse Tablet (US$), February’2026
Figure 8-47: US - Braftovi Orphan Drug Designation & Approval Year
Figure 8-48: US - Cost Per Unit & Supply of 75mg Braftovi Capsule (US$), February’2026
Figure 8-49: US - Monthly Cost of Melanoma & NSCLC Treatment with Braftovi (US$), February’2026
Figure 8-50: US - Cost Per Unit & Supply of 15mg Mektovi Tablet (US$), February’2026
Figure 8-51: US - Monthly Cost of Melanoma Treatment with Mektovi Tablet (US$), February’2026
Figure 8-52: US - Evrysdi Orphan Drug Designation & Approval Year
Figure 8-53: US - Cost Per Unit & Supply of Evrysdi (US$), February’2026
Figure 8-54: US - Hetlioz Orphan Drug Designation & Approval Year by Indication
Figure 8-55: US - Cost Per Unit & Supply of 20mg Hetlioz Capsule (US$), February’2026
Figure 8-56: US - Cost Per Unit & Supply of 4 mg/mL Hetlioz LQ Oral Suspension (US$), February’2026
Figure 8-57: Yervoy - First Approval by Indication
Figure 8-58: US - Cost Per Unit & Supply of Yervoy Solution (US$), February’2026
Figure 8-59: US - Lorbrena Orphan Drug Designation & Approval Year
Figure 8-60: US - Cost Per Unit & Supply of Lorbrena Tablet (US$), February’2026
Figure 8-61: US - Terlivaz Orphan Drug Designation & Approval Year
Figure 8-62: Terlivaz - Dosing Chart
Figure 8-63: US - Brukinsa Orphan Drug Designation & Approval Year by Indication
Figure 8-64: US - Cost Per Unit & Supply of Brukinsa 160 mg Tablet & 80 mg Capsule (US$), February’2026
Figure 8-65: US - Unit & Monthly Cost of Treatment with Brukinsa (US$), February’2026
Figure 8-66: US - Viltepso Orphan Drug Designation & Approval Year
Figure 8-67: US - Cost Per Unit & Supply of Viltepso 50mg/mL Solution (US$), February’2026
Figure 8-68: US - Eylea Orphan Drug Designation & Approval Year
Figure 8-69: US - Cost Per Unit & Supply of Eylea Solution (US$), February’2026
Figure 8-70: US - Evkeeza Orphan Drug Designation & Approval Year
Figure 8-71: US - Cost Per Unit & Supply of Evkeeza Solution (US$), February’2026
Figure 8-72: US - Sogroya Orphan Drug Designation & Approval Year
Figure 8-73: US - Cost Per Unit & Supply of Sogroya Solution (US$), February’2026
Figure 8-74: US - Skytrofa Orphan Drug Designation & Approval Year
Figure 8-75: US - Cost Per Unit & Supply of Skytrofa Injection (US$), February’2026
Figure 8-76: US - Cost Per Unit & Supply of Avastin Solution (US$), February’2026
Figure 8-77: US - Vyvgart Orphan Drug Designation & Approval Year
Figure 8-78: US - Cost Per Unit & Supply of Vyvgart & Vyvgart Hytrulo (US$), February’2026
Figure 8-79: US - Tpoxx Orphan Drug Designation & Approval Year
Figure 8-80: US - Koselugo Orphan Drug Designation & Approval Year by Indication
Figure 8-81: US - Cost Per Unit & Supply of 10mg Koselugo Capsules (US$), February’2026
Figure 8-82: US - Cost Per Unit & Supply of Koselugo 25mg Capsules (US$), February’2026
Figure 8-83: US - Cost Per Unit & Supply of 5mg and 7.5mg Koselugo Granules (US$), February’2026
Figure 8-84: US - Actemra Orphan Drug Designation & Approval Year
Figure 8-85: US - Cost Per Unit & Supply of 20 mg/mL Vials of Actemra IV Solution (US$), February’2026
Figure 8-86: US - Cost Per Unit & Supply of 162 mg/0.9 mL Actemra Prefilled Syringe (ACTpen) (US$), February’2026
Figure 8-87: US - Tecentriq Orphan Drug Designation & Approval Year
Figure 8-88: US - Cost Per Unit & Supply of Tecentriq Intravenous Solution (US$), February’2026
Figure 8-89: US - Qinlock Orphan Drug Designation & Approval Year
Figure 8-90: US - Cost Per Unit & Supply of Qinlock Tablets (US$), February’2026
Figure 8-91: US - Unit, Monthly & Annual Cost of Treatment with Qinlock (US$), February’2026
Figure 8-92: US - Pitolisant Orphan Drug Designation & Approval Year
Figure 8-93: US - Cost Per Unit & Supply of Wakix Tablets (US$), February’2026
Figure 8-94: US - Kimmtrak Orphan Drug Designation & Approval Year
Figure 8-95: US - Cost Per Unit & Supply of Kimmtrak (US$), February’2026
Figure 8-96: US - Elahere Orphan Drug Designation & Approval Year
Figure 8-97: US - Cost Per Unit & Supply of 100 mg/20 mL Elahere Solution (US$), February’2026
Figure 8-98: US - Price for Single Unit of Polivy Intravenous Powder for Injection (US$), February’2026
Figure 8-99: US - Enhertu Orphan Drug Designation & Approval Year
Figure 8-100: Enhertu - Cost of Single Treatment Cycle (US$), February’2026
Figure 8-101: US - Jakafi Orphan Drug Designation & Approval Year by Indication
Figure 8-102: US - Cost Per Unit & Supply of Different Jakafi Doses (US$), February’2026
Figure 8-103: US - Kalydeco Orphan Drug Designation & Approval Year
Figure 8-104: US - Cost Per Unit & Supply of Kalydeco Oral Granule & Tablet (US$), February’2026
Figure 8-105: US - Cost Per Unit & Supply of Symdeko Oral Tablet (US$), February’2026
Figure 8-106: US - Trikafta Orphan Drug Designation & Approval Year
Figure 8-107: US - Cost Per Unit & Supply of Trikafta (US$), February’2026
Figure 8-108: US - Orkambi Orphan Drug Designation & Approval Year
Figure 8-109: US - Cost Per Unit & Supply of Orkambi Oral Granule & Tablet (US$), February’2026
Figure 8-110: US - Hemgenix Orphan Drug Designation & Approval Year
Figure 8-111: US - Voxzogo Orphan Drug Designation & Approval Year
Figure 8-112: US - Cost Per Unit & Supply of Voxzogo Powder (US$), February’2026
Figure 8-113: US - Actimmune Orphan Drug Designation & Approval Year
Figure 8-114: US - Cost Per Unit & Supply of Actimmune Solution (US$), February’2026
Figure 8-115: US - Luspatercept Orphan Drug Designation & Approval Year
Figure 8-116: US - Cost Per Unit of Reblozyl powder (US$), February’2026
Figure 8-117: US - Monthly & Annual Cost of Treatment with Reblozyl (US$), February’2026
Figure 8-118: US - Repatha Orphan Drug Designation & Approval Year
Figure 8-119: US - Cost Per Unit & Supply of Repatha (US$), February’2026
Figure 8-120: US - Monthly & Annual Cost of Repatha Treatment in Adults with Heterozygous Familial Hypercholesterolemia (US$), February’2026
Figure 8-121: US - Rebyota Orphan Drug Designation & Approval Year
Figure 8-122: US - Cost Per Unit & Supply of Rebyota (US$), February’2026
Figure 8-123: US - Cayston Orphan Drug Designation & Approval Year
Figure 8-124: US - Cost Per Unit & Supply of Cayston Powder (US$), February’2026
Figure 8-125: US - Sylvant Orphan Drug Designation & Approval Year
Figure 8-126: US - Cost Per Unit & Supply of Sylvant Powder (US$), February’2026
Figure 8-127: US - Mylotarg Orphan Drug Designation & Approval Year
Figure 8-128: US - Besremi Orphan Drug Designation & Approval Year
Figure 8-129: US - Ravicti Orphan Drug Designation & Approval Year
Figure 8-130: US - Cost Per Unit & Supply of Ravicti (US$), February’2026
Figure 8-131: US - Cost Per Unit & Supply of Tazverik Tablet (US$), February’2026
Figure 8-132: US - Luxturna Orphan Drug Designation & Approval Year
Figure 8-133: US - Zynteglo Orphan Drug Designation & Approval Year
Figure 8-134: US - Empaveli Orphan Drug Designation & Approval Year
Figure 8-135: US - Cost Per Unit & Supply of Empaveli Solution (US$), February’2026
Figure 8-136: US - Cost of Single Dose, Monthly & Yearly Treatment with Empaveli (US$), February’2026
Figure 8-137: US - Ninlaro Orphan Drug Designation & Approval Year
Figure 8-138: US - Cost Per Unit & Supply of Ninlaro Capsule (US$), February’2026
Figure 8-139: US - Cost of Single Dose, Monthly & Yearly Treatment with Ninlaro (US$), February’2026
Figure 8-140: US - Otezla Orphan Drug Designation & Approval Year
Figure 8-141: US - Cost Per Unit & Supply of Otezla & Otezla XR Tablets (US$), February’2026
Figure 8-142: US - Arzerra Orphan Drug Designation & Approval Year
Figure 8-143: US - Cost Per Unit & Supply of Arzerra Solution (US$), February’2026
Figure 8-144: US - Macrilen Orphan Drug Designation & Approval Year
Figure 8-145: US - Tagrisso Orphan Drug Designation & Approval Year
Figure 8-146: US - Cost Per Unit & Supply of Tagrisso Tablets (US$), February’2026
Figure 8-147: US - Tagrisso Monthly, Yearly & Full Course Treatment Cost (US$), February’2026
Figure 8-148: US - Gammagard Liquid Orphan Drug Designation & Approval Year
Figure 8-149: US - Cost Per Unit & Supply of Different Gammagard Liquid Vials (US$), February’2026
Figure 8-150: US - Fintepla Orphan Drug Designation & Approval Year
Figure 8-151: US - Cost Per Unit & Supply of Fintepla Solution (US$), February’2026
Figure 8-152: US - Daybue Orphan Drug Designation & Approval Year
Figure 8-153: US - Cost Per Unit & Supply of Daybue Liquid (US$), February’2026
Figure 8-154: US - Takhzyro Orphan Drug Designation & Approval Year
Figure 8-155: US - Cost Per Unit & Supply of Takhzyro Solution (US$), February’2026
Figure 8-156: US - Cyramza Orphan Drug Designation & Approval Year
Figure 8-157: US - Cost Per Unit & Supply of Cyramza Solution (US$), February’2026
Figure 8-158: US - Hyftor Orphan Drug Designation & Approval Year
Figure 8-159: US - Cost Per Unit & Supply of Hyftor Gel (US$), February’2026
Figure 8-160: US - Prevymis Orphan Drug Designation & Approval Year
Figure 8-161: US - Cost Per Unit & Supply of Prevymis Solution (US$), February’2026
Figure 8-162: US - Cost Per Unit & Supply of Prevymis Pellets (US$), February’2026
Figure 8-163: US - Cost Per Unit & Supply of Prevymis Tablets (US$), February’2026
Figure 8-164: US - Prevymis Orphan Drug Designation & Approval Year
Figure 8-165: US - Copiktra Orphan Drug Designation & Approval Year
Figure 8-166: US - Cost Per Unit & Supply of 15 mg Copiktra Capsules (US$), February’2026
Figure 8-167: US - Tepezza Orphan Drug Designation & Approval Year
Figure 8-168: US - Mycapssa Orphan Drug Designation & Approval Year
Figure 8-169: US - Cost Per Unit & Supply of Mycapssa Capsules (US$), February’2026
Figure 8-170: US - Juxtapid Orphan Drug Designation & Approval Year
Figure 8-171: US - Cost Per Unit & Supply of Juxtapid Capsules (US$), February’2026
Figure 8-172: US - Myalept Orphan Drug Designation & Approval Year
Figure 8-173: US - Jelmyto Orphan Drug Designation & Approval Year
Figure 8-174: US - Mitosol Orphan Drug Designation & Approval Year
Figure 8-175: US - Cost Per Unit & Supply of Mitosol Kit (US$), February’2026
Figure 8-176: US - Empliciti Orphan Drug Designation & Approval Year
Figure 8-177: US - Cost Per Unit & Supply of Empliciti Powders (US$), February’2026
Figure 8-178: US - Kcentra Orphan Drug Designation & Approval Year
Figure 8-179: US - Kyprolis Orphan Drug Designation & Approval Year by Indication
Figure 8-180: US - Cost Per supply of 10mg, 30mg & 60mg Kyprolis (US$), February’2026
Figure 8-181: US - Enspryng Orphan Drug Designation & Approval Year by Indications
Figure 8-182: US - Sirturo Orphan Drug Designation & Approval Year by Indications
Figure 8-183: US - Cost Per Unit & Supply of 20mg & 100mg Sirturo Tablets (US$), February’2026
Figure 8-184: US - Tavalisse Orphan Drug Designation & Approval Year by Indications
Figure 8-185: US - Cost Per Unit & Supply of Tavalisse Tablets (US$), February’2026
Figure 8-186: US - Gleolan Orphan Drug Designation & Approval Year by Indications
Figure 8-187: US - Keveyis Orphan Drug Designation & Approval Year
Figure 8-188: US - Cost Per Unit & Supply of Keveyis Tablets (US$), February’2026
Figure 8-189: US - Zolgensma Orphan Drug Designation & Approval Year
Figure 8-190: US - Darzalex Orphan Drug Designation & Approval Year
Figure 8-191: US - Cost Per Unit & Supply of Darzalex Solution (US$), February’2026
Figure 8-192: US - Darzalex Orphan Drug Designation & Approval Year
Figure 8-193: US - Cost Per Unit & Supply of Darzalex Faspro Solution (US$), February’2026
Figure 8-194: US - Doptelet Orphan Drug Designation & Approval Year
Figure 8-195: US - Cost Per Unit & Supply of Doptelet Tablet (US$), February’2026
Figure 8-196: US - Endari Orphan Drug Designation & Approval Year
Figure 8-197: US - Cost Per Unit & Supply of Endari Powder (US$), February’2026
Figure 8-198: US - NutreStore Orphan Drug Designation & Approval Year
Figure 8-199: US - Cost Per Unit & Supply of NutreStore (US$), February’2026
Figure 8-200: US - Lymphoseek Orphan Drug Designation & Approval Year
Figure 8-201: US - Krystexxa Orphan Drug Designation & Approval Year
Figure 8-202: US - Cost Per Unit & Supply of Krystexxa (US$), February’2026
Figure 8-203: US - Krystexxa Orphan Drug Designation & Approval Year
Figure 8-204: US - Cost Per Unit & Supply of Korlym (US$), February’2026
Figure 8-205: US - Kalbitor Orphan Drug Designation & Approval Year
Figure 8-206: US - Cost Per Unit & Supply of Kalbitor (US$), February’2026
Figure 8-207: US - Livtencity Orphan Drug Designation & Approval Year
Figure 8-208: US - Cost Per Unit & Supply of Livtencity (US$), February’2026
Figure 8-209: US - Enjaymo Orphan Drug Designation & Approval Year
Figure 8-210: US - Cost Per Unit & Supply of Enjaymo (US$), February’2026
Figure 8-211: US - Rethymic Orphan Drug Designation & Approval Year
Figure 8-212: US - Coartem Orphan Drug Designation & Approval Year
Figure 8-213: US - Cost Per Unit & Supply of Coartem (US$), February’2026
Figure 8-214: US - Nulibry Orphan Drug Designation & Approval Year
Figure 8-215: US - Inmazeb Orphan Drug Designation & Approval Year
Figure 8-216: US - Valtoco Orphan Drug Designation & Approval Year
Figure 8-217: US - Cost Per Unit & Supply of Valtoco (US$), February’2026
Figure 8-218: US - Detectnet Orphan Drug Designation & Approval Year
Figure 8-219: US - Folotyn Orphan Drug Designation & Approval Year
Figure 8-220: US - Cost Per Unit & Supply of Folotyn (US$), February’2026
Figure 8-221: US - Cystadane Orphan Drug Designation & Approval Year
Figure 8-222: US - Cost Per Unit & Supply of Cystadane (US$), February’2026
Figure 8-223: US - Cystaran Orphan Drug Designation & Approval Year
Figure 8-224: US - Cost Per Unit & Supply of Cystaran (US$), February’2026
Figure 8-225: US - Voraxaze Orphan Drug Designation & Approval Year
Figure 8-226: US - Cost of Treatment with Voraxaze (US$), February’2026
Figure 8-227: US - Cost Per Unit & Supply of Praxbind (US$), February’2026
Figure 8-228: US - Mepron Orphan Drug Designation & Approval Year
Figure 8-229: US - Cost Per Unit & Supply of Mepron (US$), February’2026
Figure 8-230: US - Vistogard Orphan Drug Designation & Approval Year
Figure 8-231: US - Cost Per Unit & Supply of Vistogard (US$), February’2026
Figure 8-232: US - Xuriden Orphan Drug Designation & Approval Year
Figure 8-233: US - Cost Per Unit & Supply of Mepron (US$), February’2026
Figure 8-234: US - Xatmep Orphan Drug Designation & Approval Year
Figure 8-235: US - Cost Per Unit & Supply of Xatmep (US$), February’2026
Figure 8-236: US - Purixan Orphan Drug Designation & Approval Year
Figure 8-237: US - Cost Per Unit & Supply of Purixan (US$), February’2026
Figure 8-238: US - Ascor Orphan Drug Designation & Approval Year
Figure 8-239: US - Cost Per Unit & Supply of Ascor (US$), February’2026
Figure 8-240: US - Tepadina Orphan Drug Designation & Approval Year
Figure 8-241: US - Cost of Per supply of Tepadina (US$), February’2026
Figure 8-242: US - BAT Orphan Drug Designation & Approval Year
Figure 8-243: US - Busulfex Orphan Drug Designation & Approval Year
Figure 8-244: US - Cost Per Unit & Supply of Busulfex (US$), February’2026
Figure 8-245: US - Lipiodol Orphan Drug Designation & Approval Year
Figure 8-246: US - Cost Per Unit & Supply of Lipiodol (US$), February’2026
Figure 8-247: US - Doxil Orphan Drug Designation & Approval Year
Figure 8-248: US - Cost Per Unit & Supply of Doxil (US$), February’2026
Figure 8-249: US - Nymalize Orphan Drug Designation & Approval Year
Figure 8-250: US - Cost Per Unit & Supply of Nymalize (US$), February’2026
Figure 8-251: US - Priftin Orphan Drug Designation & Approval Year
Figure 8-252: US - Cost Per Unit & Supply of Priftin (US$), February’2026
Figure 8-253: US - Gralise Orphan Drug Designation & Approval Year
Figure 8-254: US - Cost Per Unit & Supply of Gralise (US$), February’2026
Figure 8-255: US - Gralise Orphan Drug Designation & Approval Year
Figure 8-256: US - Cost Per Unit & Supply of Galzin (US$), February’2026
Figure 8-257: US - NeoProfen Orphan Drug Designation & Approval Year
Figure 8-258: US - Cost Per Unit & Supply of NeoProfen (US$), February’2026
Figure 8-259: US - Photofrin Orphan Drug Designation & Approval Year
Figure 8-260: US - Retisert Orphan Drug Designation & Approval Year
Figure 8-261: US - Elmiron Orphan Drug Designation & Approval Year
Figure 8-262: US - Cost Per Unit & Supply of Elmiron (US$), February’2026
Figure 8-263: US - Cost Per Unit & Supply of Extraneal (US$), February’2026
Figure 8-264: US - Azedra Orphan Drug Designation & Approval Year
Figure 8-265: US - Elelyso Orphan Drug Designation & Approval Year
Figure 8-266: US - Tecentriq Orphan Drug Designation & Approval Year
Figure 8-267: US - Cost Per Unit & Supply of Pedmark Intravenous Solution (US$), February’2026
Figure 8-268: US - Palynziq Orphan Drug Designation & Approval Year
Figure 8-269: US - Cost Per Unit & Supply of 2.5mg & 10mg Palynziq Subcutaneous Solutions (US$), February’2026
Figure 8-270: US - Cost Per Unit & Supply of 20mg Palynziq Subcutaneous Solution (US$), February’2026
Figure 8-271: US - Spinraza Orphan Drug Designation & Approval Year
Figure 8-272: US - Cost Per Unit & Supply of Spinraza (US$), February’2026
Figure 8-273: US - Cost of Treatment With Spinraza (US$), February’2026
Figure 8-274: US - Cost of Treatment With Elzonris (US$), February’2026
Figure 8-275: US - Cost Per Unit & Supply of Rituxan (US$), February’2026
Figure 8-276: US - Rezlidhia Orphan Drug Designation & Approval Year
Figure 8-277: US - Cost Per Unit & Supply of Rezlidhia (US$), February’2026
Figure 8-278: US - Cost of Treatment With Rezlidhia (US$), February’2026
Figure 8-279: US - Triptodur Orphan Drug Designation & Approval Year
Figure 8-280: US - Tecartus Orphan Drug Designation & Approval Year
Figure 8-281: US - Cresemba Orphan Drug Designation & Approval Year
Figure 8-282: US - Cost Per Unit & Supply of Cresemba (US$), February’2026
Figure 8-283: US - Opsumit Orphan Drug Designation & Approval Year
Figure 8-284: US - Cost Per Unit & Supply of Opsumit (US$), February’2026
Figure 8-285: US - Cost of Treatment With Opsumit (US$), February’2026
Figure 8-286: US - Uplizna Orphan Drug Designation & Approval Year
Figure 8-287: US - Cost Per Unit & Supply of Uplizna (US$), February’2026
Figure 8-288: US - Givlaari Orphan Drug Designation & Approval Year
Figure 8-289: US - Cost of Treatment With Givlaari (US$), February’2026
Figure 8-290: US - Oxlumo Orphan Drug Designation & Approval Year
Figure 8-291: US - Cost Per Unit & Supply of Oxlumo (US$), February’2026
Figure 8-292: US - Velcade Orphan Drug Designation & Approval Year
Figure 8-293: US - Exondys 51 Orphan Drug Designation & Approval Year
Figure 8-294: US - Cost Per Unit & Supply of Exondys 51 (US$), February’2026
Figure 8-295: US - Vyondys 53 Orphan Drug Designation & Approval Year
Figure 8-296: US - Cost Per Unit & Supply of Vyondys 53 (US$), February’2026
Figure 8-297: US - Amondys 45 Orphan Drug Designation & Approval Year
Figure 8-298: US - Cost Per Unit & Supply of Amondys 45 (US$), February’2026
Figure 8-299: US - Besponsa Orphan Drug Designation & Approval Year
Figure 8-300: US - Epidiolex Orphan Drug Designation & Approval Year
Figure 8-301: US - Cost Per Unit & Supply of Epidiolex (US$), February’2026
Figure 8-302: US - Xyrem Orphan Drug Designation & Approval Year
Figure 8-303: US - Cost Per Unit & Supply of Xyrem (US$), February’2026
Figure 8-304: US - Xywav Orphan Drug Designation & Approval Year
Figure 8-305: US - Cost Per Unit & Supply of Xywav (US$), February’2026
Figure 8-306: US - Vyxeos Orphan Drug Designation & Approval Year
Figure 8-307: US - Cost Per Unit & Supply of Vyxeos (US$), February’2026
Figure 8-308: US - Unituxin Orphan Drug Designation & Approval Year
Figure 8-309: US - Cost Per Unit & Supply of Unituxin (US$), February’2026
Figure 8-310: US - Alimta Orphan Drug Designation & Approval Year
Figure 8-311: US - Cost of supply of Alimta (US$), February’2026
Figure 8-312: US - Aliqopa Orphan Drug Designation & Approval Year
Figure 8-313: US - Cost of Treatment With Aliqopa (US$), February’2026
Figure 8-314: US - Aliqopa Orphan Drug Designation & Approval Year
Figure 8-315: US - Cost Per Unit & Supply of Oncaspar (US$), February’2026
Figure 8-316: US - Rylaze Orphan Drug Designation & Approval Year
Figure 8-317: US - Cost Per Unit & of supply of Rylaze (US$), February’2026
Figure 8-318: US - Vonjo Orphan Drug Designation & Approval Year
Figure 8-319: US - Cost Per Unit & of supply of Vonjo (US$), February’2026
Figure 8-320: US - Pyrukynd Orphan Drug Designation & Approval Year
Figure 8-321: US - Cost Per Unit & Supply of Pyrukynd (US$), February’2026
Figure 8-322: US - Uptravi Orphan Drug Designation & Approval Year
Figure 8-323: US - Cost of supply of Uptravi Intravenous Injection (US$), February’2026
Figure 8-324: US - Cost Per Unit & Supply of 200mcg Uptravi Oral Tablets (US$), February’2026
Figure 8-325: US - Cost Per Unit & Supply of 400mcg, 600mcg, 800mcg, 000mcg, 1200mcg, 1400mcg & 1600mcg Uptravi Oral Tablets (US$), February’2026
Figure 8-326: US - Xermelo Orphan Drug Designation & Approval Year
Figure 8-327: US - Cost Per Unit & Supply of Xermelo (US$), February’2026
Figure 8-328: US - Gilotrif Orphan Drug Designation & Approval Year
Figure 8-329: US - Cost Per Unit & Supply of Gilotrif Oral Tablets (US$), February’2026
Figure 8-330: US - Wilate Orphan Drug Designation & Approval Year
Figure 8-331: US - Stivarga Orphan Drug Designation & Approval Year
Figure 8-332: US - Cost Per Unit & Supply of Stivarga Oral Tablets (US$), February’2026
Figure 8-333: US - Alkindi Sprinkle Orphan Drug Designation & Approval Year
Figure 8-334: US - Cost Per Unit & Supply of Alkindi Sprinkle (US$), February’2026
Figure 8-335: US - Gamifant Orphan Drug Designation & Approval Year
Figure 8-336: US - Cost Per Unit & Supply of Gamifant (US$), February’2026
Figure 8-337: US - Turalio Orphan Drug Designation & Approval Year
Figure 8-338: US - Cost Per Unit & Supply of Turalio (US$), February’2026
Figure 8-339: US - Yondelis Orphan Drug Designation & Approval Year
Figure 8-340: US - Cost per Yondelis Injection (US$), February’2026
Figure 8-341: US - Yondelis Orphan Drug Designation & Approval Year
Figure 8-342: US - Rebinyn Orphan Drug Designation & Approval Year
Figure 8-343: US - Onureg Orphan Drug Designation & Approval Year
Figure 8-344: US - Cost Per Unit & Supply of Onureg (US$), February’2026
Figure 8-345: US - Cost per Onureg Cycle (US$), February’2026
Figure 8-346: US - Cabometyx Orphan Drug Designation & Approval Year
Figure 8-347: US - Cost Per Unit & Supply of Cabometyx (US$), February’2026
Figure 8-348: US - Cabometyx Orphan Drug Designation & Approval Year
Figure 8-349: US - Cost Per Unit & Supply of Cometriq (US$), February’2026
Figure 8-350: US - Orenitram Orphan Drug Designation & Approval Year
Figure 8-351: US - Cost Per Unit & Supply of 0.125 mg-0.25 mg & 0.125 mg-0.25 mg-1 mg Orenitram Tablets (US$), February’2026
Figure 8-352: US - Cost Per Unit & Supply of Orenitram 10-Tablets Supplies of Varying Doses (US$), February’2026
Figure 8-353: US - Cost Per Unit & Supply of Orenitram 100-Tablets Supplies of Varying Doses (US$), February’2026
Figure 8-354: US - Remodulin Orphan Drug Designation & Approval Year
Figure 8-355: US - Cost Per Unit & Supply of Remodulin Injection (US$), February’2026
Figure 8-356: US - Tavneos Orphan Drug Designation & Approval Year
Figure 8-357: US - Cost Per Unit & Supply of Tavneos (US$), February’2026
Figure 8-358: US - Impavido Orphan Drug Designation & Approval Year
Figure 8-359: US - Cost Per Unit & Supply of Impavido (US$), February’2026
Figure 8-360: US - Carvykti Orphan Drug Designation & Approval Year
Figure 8-361: US - Xiaflex Orphan Drug Designation & Approval Year
Figure 8-362: US - Hemlibra Orphan Drug Designation & Approval Year
Figure 8-363: US - Cost of supply of Hemlibra (US$), February’2026
Figure 8-364: US - Recorlev Orphan Drug Designation & Approval Year
Figure 8-365: US - Cost per Unit & supply of Recorlev (US$), February’2026
Figure 8-366: US - Exkivity Orphan Drug Designation & Approval Year
Figure 8-367: US - Cost per Unit & supply of Exkivity (US$), February’2026
Figure 8-368: US - Kymriah Orphan Drug Designation & Approval Year
Figure 8-369: US - Brineura Orphan Drug Designation & Approval Year
Figure 8-370: US - Monthly & Yearly Cost of Treatment With Brineura (US$), February’2026
Figure 8-371: US - Istodax Orphan Drug Designation & Approval Year
Figure 8-372: US - Bosulif Orphan Drug Designation & Approval Year
Figure 8-373: US - Cost per Unit & supply of Bosulif Oral Tablets (US$), February’2026
Figure 8-374: US - Cost per Unit & supply of Bosulif Oral Capsules (US$), February’2026
Figure 8-375: US - Idhifa Orphan Drug Designation & Approval Year
Figure 8-376: US - Monthly & Yearly Cost of Treatment With Idhifa (US$), February’2026
Figure 8-377: US - Arcalyst Orphan Drug Designation & Approval Year
Figure 8-378: US - Cost Per Unit & Supply of Arcalyst (US$), February’2026
Figure 8-379: US - Xgeva Orphan Drug Designation & Approval Year
Figure 8-380: US - Cost Per Unit & Supply of Xgeva (US$), February’2026
Figure 8-381: US - Dojolvi Orphan Drug Designation & Approval Year
Figure 8-382: US - Cost Per Unit & Supply of Dojolvi (US$), February’2026
Figure 8-383: US - Dojolvi Orphan Drug Designation & Approval Year
Figure 8-384: US - Cost Per Unit & Supply of Pomalyst (US$), February’2026
Figure 8-385: US - Cost Per Unit & Supply of Gleevec (US$), February’2026
Figure 8-386: US - Nexavar Orphan Drug Designation & Approval Year
Figure 8-387: US - Cost Per Unit & Supply of Nexavar (US$), February’2026
Figure 8-388: US - Nexavar Orphan Drug Designation & Approval Year
Figure 8-389: US - Cost of Supply of Treanda (US$), February’2026
Figure 8-390: US - Nexavar Orphan Drug Designation & Approval Year
Figure 8-391: US - Cost Per Unit & Supply of Treanda (US$), February’2026
Figure 8-392: US - Botox Orphan Drug Designation & Approval Year
Figure 8-393: US - Cost of Supply of Botox (US$), February’2026
Figure 8-394: US - Orfadin Orphan Drug Designation & Approval Year
Figure 8-395: US - Cost Per Unit & Supply of Orfadin Capsules (US$), February’2026
Figure 8-396: US - Cost Per Unit & Supply of Orfadin Suspension (US$), February’2026
Figure 8-397: US - Promacta Orphan Drug Designation & Approval Year
Figure 8-398: US - Cost Per Unit & Supply of 12.5mg & 25mg Promacta Tablets (US$), February’2026
Figure 8-399: US - Cost Per Unit & Supply of 50mg Promacta Tablet (US$), February’2026
Figure 8-400: US - Cost Per Unit & Supply of Promacta Powder (US$), February’2026
Figure 8-401: US - Eloctate Orphan Drug Designation & Approval Year
Figure 8-402: US - Carbaglu Orphan Drug Designation & Approval Year
Figure 8-403: US - Cost Per Unit & Supply of Carbaglu (US$), February’2026
Figure 8-404: US - Xospata Orphan Drug Designation & Approval Year
Figure 8-405: US - Cost Per Unit & Supply of Xospata (US$), February’2026
Figure 8-406: US - Defitelio Orphan Drug Designation & Approval Year
Figure 8-407: US - Cost Per Unit & Supply of Defitelio (US$), February’2026
Figure 8-408: US - Thalomid Orphan Drug Designation & Approval Year
Figure 8-409: US - Cost Per Unit & Supply of 50mg Thalomid Capsule (US$), February’2026
Figure 8-410: US - Cost Per Unit & Supply of 100mg & 150mg Thalomid Capsule (US$), February’2026
Figure 8-411: US - Agrylin Orphan Drug Designation & Approval Year
Figure 8-412: US - Cost Per Unit & Supply of Agrylin (US$), February’2026
Figure 8-413: US - Xyntha Orphan Drug Designation & Approval Year
Figure 8-414: US - Valchlor Orphan Drug Designation & Approval Year
Figure 8-415: US - Cost Per Unit & Supply of Valchlor (US$), February’2026
Figure 8-416: US - Antizol Orphan Drug Designation & Approval Year
Figure 8-417: US - Rozlytrek Orphan Drug Designation & Approval Year
Figure 8-418: US - Cost Per Unit & Supply of Rozlytrek Oral Capsule (US$), February’2026
Figure 8-419: US - Cost Per Unit & Supply of Rozlytrek Oral Pellet (US$), February’2026
Figure 8-420: US - Poteligeo Orphan Drug Designation & Approval Year
Figure 8-421: US - Cost Per Unit & Supply of Poteligeo (US$), February’2026
Figure 8-422: US - Cost Per Unit & Supply of Humira (US$), February’2026
Figure 8-423: US - Kanuma Orphan Drug Designation & Approval Year
Figure 8-424: US - Cost Per Unit & Supply of Kanuma (US$), February’2026
Figure 8-425: US - Sensipar Orphan Drug Designation & Approval Year
Figure 8-426: US - Cost Per Unit & Supply of Sensipar (US$), February’2026
Figure 8-427: US - Gattex Orphan Drug Designation & Approval Year
Figure 8-428: US - Crysvita Orphan Drug Designation & Approval Year
Figure 8-429: US - Cost Per Unit & Supply of Crysvita (US$), February’2026
Figure 8-430: US - Serostim Orphan Drug Designation & Approval Year
Figure 8-431: US - Cost Per Unit & Supply of Serostim (US$), February’2026
Figure 8-432: US - Zorbtive Orphan Drug Designation & Approval Year
Figure 8-433: US - Cost Per Unit & Supply of Zorbtive (US$), February’2026
Figure 8-434: US - Caprelsa Orphan Drug Designation & Approval Year
Figure 8-435: US - Cost Per Unit & Supply of Caprelsa (US$), February’2026
Figure 8-436: US - Zolinza Orphan Drug Designation & Approval Year
Figure 8-437: US - Cost Per Unit & Supply of Zolinza (US$), February’2026
Figure 8-438: US - Vitrakvi Orphan Drug Designation & Approval Year
Figure 8-439: US - Cost Per Unit & Supply of Vitrakvi Capsule (US$), February’2026
Figure 8-440: US - Cost Per Unit & Supply of Vitrakvi Solution (US$), February’2026
Figure 8-441: US - Obizur Orphan Drug Designation & Approval Year
Figure 8-442: US - Obizur Orphan Drug Designation & Approval Year
Figure 8-443: US - Torisel Orphan Drug Designation & Approval Year
Figure 8-444: US - Unit & Monthly Cost of Treatment With Torisel (US$), February’2026
Figure 8-445: US - Nayzilam Orphan Drug Designation & Approval Year
Figure 8-446: US - Cost Per Unit & Supply of Nayzilam (US$), February’2026
Figure 8-447: US - Zokinvy Orphan Drug Designation & Approval Year
Figure 8-448: US - Cost Per Unit & Supply of Zokinvy (US$), February’2026
Figure 8-449: Zokinvy - Recommended Dosage and Administration for 115 mg/m2 Body Surface Area-Based Dosing
Figure 8-450: US - Ayvakit Orphan Drug Designation & Approval Year
Figure 8-451: US - Cost Per Unit & Supply of Ayvakit (US$), February’2026
Figure 8-452: US - Mekinist Orphan Designations & Approval Year
Figure 8-453: US - Cost Per Unit & Supply of Mekinist Oral Tablet (US$), February’2026
Figure 8-454: US - Cost Per Unit & Supply of Mekinist Powder (US$), February’2026
Figure 8-455: US - Praluent Orphan Drug Designation & Approval Year
Figure 8-456: US - Cost Per Unit & Supply of Praluent (US$), February’2026
Figure 8-457: US - Yescarta Orphan Drug Designation & Approval Year
Figure 8-458: US - Xifaxan Orphan Drug Designation & Approval Year
Figure 8-459: US - Cost Per Unit & Supply of Xifaxan (US$), February’2026
Figure 8-460: US - Lunsumio Orphan Drug Designation & Approval Year
Figure 8-461: US - Cost Per Unit & Supply of Lunsumio (US$), February’2026
Figure 8-462: US - Bylvay Orphan Drug Designation & Approval Year
Figure 8-463: US - Cost Per Unit & Supply of Bylvay Oral Capsule (US$), February’2026
Figure 8-464: US - Cost Per Unit & Supply of Bylvay Oral Pellet (US$), February’2026
Figure 8-465: US - Tarpeyo Orphan Drug Designation & Approval Year
Figure 8-466: US - Cost Per Unit & Supply of Tarpeyo (US$), February’2026
Figure 8-467: US - Ultomiris Orphan Drug Designation & Approval Year
Figure 8-468: US - Cost Per Unit & Supply of Ultomiris (US$), February’2026
Figure 8-469: US - Cuvrior Orphan Drug Designation & Approval Year
Figure 8-470: US - Cost Per Unit & Supply of Cuvrior (US$), February’2026
Figure 8-471: US - Bavencio Orphan Drug Designation & Approval Year
Figure 8-472: US - Cost Per Unit & Supply of Bavencio (US$), February’2026
Figure 8-473: US - Pemazyre Orphan Drug Designation & Approval Year
Figure 8-474: US - Cost Per Unit & Supply of Pemazyre (US$), February’2026
Figure 8-475: US - Relyvrio Orphan Drug Designation & Approval Year
Figure 8-476: US - Cost Per Unit & Supply of Relyvrio (US$), February’2026
Figure 8-477: US - Inqovi Orphan Drug Designation & Approval Year
Figure 8-478: US - Cost Per Unit & Supply of Inqovi (US$), February’2026
Figure 8-479: US - HepaGam B Orphan Drug Designation & Approval Year
Figure 8-480: US - Cost Per Unit & Supply of HepaGam B (US$), February’2026
Figure 8-481: US - VariZIG Orphan Drug Designation & Approval Year
Figure 8-482: US - Cost Per Unit & Supply of VariZIG (US$), February’2026
Figure 8-483: US - Camzyos Orphan Drug Designation & Approval Year
Figure 8-484: US - Cost Per Unit & Supply of Camzyos (US$), February’2026
Figure 8-485: Camzyos - Recommended Dosage for Initiation Phase
Figure 8-486: Camzyos - Recommended Dose for Maintenance Phase
Figure 8-487: US - CytoGam Orphan Drug Designation & Approval Year
Figure 8-488: US - Cost Per Unit & Supply of CytoGam (US$), February’2026
Figure 8-489: US - Letairis Orphan Drug Designation & Approval Year
Figure 8-490: US - Cost Per Unit & Supply of Letairis (US$), February’2026
Figure 8-491: US - Plerixafor Orphan Drug Designation & Approval Year
Figure 8-492: US - Cost Per Unit & Supply of Mozobil (US$), February’2026
Figure 8-493: US - Inotersen Orphan Drug Designation & Approval Year
Figure 8-494: US - Cost Per Unit & Supply of Tegsedi (US$), February’2026
Figure 8-495: US - Tetrabenazine Orphan Drug Designation & Approval Year
Figure 8-496: US - Cost Per Unit & Supply of Xenazine (US$), February’2026
Figure 8-497: US - Mecasermin Orphan Drug Designation & Approval Year
Figure 8-498: US - Cost Per Unit & Supply of Increlex (US$), February’2026
Figure 8-499: US - Mecasermin Orphan Drug Designation & Approval Year
Figure 8-500: US - Cost Per Unit & Supply of Ethyol (US$), February’2026
Figure 8-501: US - Tacrolimus Extended Release Orphan Drug Designation & Approval Year
Figure 8-502: US - Cost Per Unit & Supply of Astagraf XL (US$), February’2026
Figure 8-503: US - Cost Per Unit & Supply of Prograf Intravenous Solution (US$), February’2026
Figure 8-504: US - Cost Per Unit & Supply of Prograf Oral Capsule (US$), February’2026
Figure 8-505: US - Cost Per Unit & Supply of Prograf Oral Granule (US$), February’2026
Figure 8-506: US - Ztalmy Orphan Drug Designation & Approval Year
Figure 8-507: US - Cost Per Unit & Supply of Ztalmy (US$), February’2026
Figure 8-508: US - Cost Per Unit & Supply of Keytruda (US$), February’2026
Figure 8-509: US - Cost Per Unit & Supply of 10 mg-50 mg-100 mg Venclexta (US$), February’2026
Figure 8-510: US - Cost Per Unit & Supply of 10 mg Venclexta (US$), February’2026
Figure 8-511: US - Cost Per Unit & Supply of 50 mg Venclexta (US$), February’2026
Figure 8-512: US - Cost Per Unit & Supply of 100 mg Venclexta (US$), February’2026
Figure 8-513: US - Beleodaq Orphan Drug Designation & Approval Year
Figure 8-514: US - Radicava Orphan Drug Designation & Approval Year
Figure 8-515: US - Cost Per Unit & Supply of Radicava & Radicava ORS (US$), February’2026
Figure 8-516: US - Jaypirca Orphan Drug Designation & Approval Year
Figure 8-517: US - Cost Per Unit & Supply of Jaypirca (US$), February’2026
Figure 8-518: US - Filspari Orphan Drug Designation & Approval Year
Figure 8-519: US - Cost Per Unit & Supply of Filspari (US$), February’2026
Figure 8-520: US - Tibsovo Orphan Drug Designation & Approval Year
Figure 8-521: US - Cost Per Unit & Supply of Filspari (US$), February’2026
Figure 8-522: US - Joenja Orphan Drug Designation & Approval Year
Figure 8-523: US - Cost Per Unit & Supply of Joenja (US$), February’2026
Figure 8-524: US - Idecabtagene Vicleucel Orphan Drug Designation & Approval Year
Figure 8-525: US - Spevigo Orphan Drug Designation & Approval Year
Figure 8-526: US - Cost Per Unit & Supply of Spevigo (US$), February’2026
Figure 8-527: US - Apokyn Orphan Drug Designation & Approval Year
Figure 8-528: US - Cost Per Unit & Supply of Apokyn (US$), February’2026
Figure 8-529: US - Northera Orphan Drug Designation & Approval Year
Figure 8-530: US - Cost Per Unit & Supply of Northera (US$), February’2026
Figure 8-531: US - Qutenza Orphan Drug Designation & Approval Year
Figure 8-532: US - Infasurf Orphan Drug Designation & Approval Year
Figure 8-533: US - Cost Per Unit & Supply of Infasurf (US$), February’2026
Figure 8-534: US - Jynarque Orphan Drug Designation & Approval Year
Figure 8-535: US - Cost Per Unit & Supply of Jynarque (US$), February’2026
Figure 8-536: US - Ivabradine Orphan Drug Designation & Approval Year
Figure 8-537: US - Cost Per Unit & Supply of Corlanor (US$), February’2026
Figure 8-538: US - Abraxane Orphan Drug Designation & Approval Year
Figure 8-539: US - Pirfenidone Orphan Drug Designation & Approval Year
Figure 8-540: US - Cost Per Unit & Supply of Esbriet (US$), February’2026
Figure 8-541: US - Albutrepenonacog Alfa Orphan Drug Designation & Approval Year
Figure 8-542: US - Tobramycin for Inhalation Orphan Drug Designation & Approval Year
Figure 8-543: US - Cost Per Unit & Supply of TOBI (US$), February’2026
Figure 8-544: US - Vestronidase Alfa Drug Designation & Approval Year
Figure 8-545: US - Cost Per Unit & Supply of Mepsevii (US$), February’2026
Figure 8-546: US - Sildenafil Orphan Drug Designation & Approval Year
Figure 8-547: US - Cost Per Unit & Supply of Revatio (US$), February’2026
Figure 8-548: US - Osilodrostat Orphan Drug Designation & Approval Year
Figure 8-549: US - Cost Per Unit & Supply of Isturisa 1mg (US$), February’2026
Figure 8-550: US - Cost Per Unit & Supply of Isturisa 5mg (US$), February’2026
Figure 8-551: US - Caplacizumab Orphan Drug Designation & Approval Year
Figure 8-552: US - Cysteamine Orphan Drug Designation & Approval Year
Figure 8-553: US - Cost Per Unit & Supply of Cystagon (US$), February’2026
Figure 8-554: US - Catridecacog Orphan Drug Designation & Approval Year
Figure 8-555: US - Alglucosidase Alfa Orphan Drug Designation & Approval Year
Figure 8-556: US - Alglucosidase Alfa Orphan Drug Designation & Approval Year
Figure 8-557: US - Cost Per Unit & Supply of Vimizim (US$), February’2026
Figure 8-558: US - Sabril Orphan Drug Designation & Approval Year
Figure 8-559: US - Cost Per Unit & Supply of Sabril (US$), February’2026
Figure 8-560: US - Parathyroid Hormone Orphan Drug Designation & Approval Year
Figure 8-561: US - Cost Per Unit & Supply of Natpara (US$), February’2026
Figure 8-562: US - Ciclosporin Orphan Drug Designation & Approval Year
Figure 8-563: US - Cost Per Unit & Supply of Verkazia (US$), February’2026
Figure 8-564: US - Ciclosporin Orphan Drug Designation & Approval Year
Figure 8-565: US - Cost Per Unit & Supply of Verkazia (US$), February’2026
Figure 8-566: US - Arranon Orphan Drug Designation & Approval Year
Figure 8-567: US - Cost Per Unit & Supply of Arranon (US$), February’2026
Figure 8-568: US - Cost Per Unit & Supply of Ixiaro (US$), February’2026
Figure 8-569: US - Norditropin Orphan Drug Designation & Approval Year
Figure 8-570: US - Cost Per Unit & Supply of Norditropin FlexPro (US$), February’2026
Figure 8-571: US - Dysport Orphan Drug Designation & Approval Year
Figure 8-572: US - Cost Per Unit & Supply of Dysport (US$), February’2026
Figure 8-573: US - Xenpozyme Orphan Drug Designation & Approval Year
Figure 8-574: US - Cost Per Unit & Supply of Xenpozyme (US$), February’2026
Figure 8-575: US - Rufinamide Orphan Drug Designation & Approval Year
Figure 8-576: US - Cost Per Unit & Supply of Banzel Oral Suspension (US$), February’2026
Figure 8-577: US - Cost Per Unit & Supply of Banzel Oral Tablet (US$), February’2026
Figure 8-578: US - Laronidase Orphan Drug Designation & Approval Year
Figure 8-579: US - Cost Per Unit & Supply of Aldurazyme (US$), February’2026
Figure 8-580: US - Bexarotene Orphan Drug Designation & Approval Year
Figure 8-581: US - Cost Per Unit & Supply of Targretin (US$), February’2026
Figure 8-582: US - Fidaxomicin Orphan Drug Designation & Approval Year
Figure 8-583: US - Cost Per Unit & Supply of Dificid (US$), February’2026
Figure 8-584: US - Clobazam Orphan Drug Designation & Approval Year
Figure 8-585: US - Cost Per Unit & Supply of Onfi (US$), February’2026
Figure 8-586: US - Raloxifene Orphan Drug Designation & Approval Year
Figure 8-587: US - Cost Per Unit & Supply of Evista (US$), February’2026
Figure 8-588: US - Rasburicase Orphan Drug Designation & Approval Year
Figure 8-589: US - Cost Per Unit & Supply of Elitek (US$), February’2026
Figure 8-590: US - Gamunex-C Orphan Drug Designation & Approval Year
Figure 8-591: US - Cost Per Unit & Supply of Gamunex-C (US$), February’2026
Figure 8-592: US - Brigatinib Orphan Drug Designation & Approval Year
Figure 8-593: US - Cost Per Unit & Supply of Alunbrig (US$), February’2026
Figure 8-594: US - Noxafil Orphan Drug Designation & Approval Year
Figure 8-595: US - Cost Per Unit & Supply of Noxafil (US$), February’2026
Figure 8-596: US - Dacomitinib Orphan Drug Designation & Approval Year
Figure 8-597: US - Cost Per Unit & Supply of Vizimpro (US$), February’2026
Figure 8-598: US - Envarsus XR Orphan Drug Designation & Approval Year
Figure 8-599: US - Cost Per Unit & 30-Tablet Supply of Envarsus XR (US$), February’2026
Figure 8-600: US - Cost Per Unit & 100-Tablet Supply of Envarsus XR (US$), February’2026
Figure 8-601: US - Albendazole Orphan Drug Designation & Approval Year
Figure 8-602: US - Cost Per Unit & Supply of Albenza (US$), February’2026
Figure 8-603: US - Netspot Orphan Drug Designation & Approval Year
Figure 8-604: US - Cost Per Unit & Supply of Trisenox (US$), February’2026
Figure 8-605: US - Buphenyl Orphan Drug Designation & Approval Year
Figure 8-606: US - Cost Per Unit & Supply of Buphenyl (US$), February’2026
Figure 8-607: US - Ammonul Orphan Drug Designation & Approval Year
Figure 8-608: US - Cost Per Unit & Supply of Ammonul (US$), February’2026
Figure 8-609: US - Elapegademase Orphan Drug Designation & Approval Year
Figure 8-610: US - Cost Per Unit & Supply of Revcovi (US$), February’2026
Figure 8-611: US - RiaSTAP Orphan Drug Designation & Approval Year
Figure 8-612: US - Elapegademase Orphan Drug Designation & Approval Year
Figure 8-613: US - Cost Per Unit & Supply of Provigil (US$), February’2026
Figure 8-614: US - Glycopyrrolate Orphan Drug Designation & Approval Year
Figure 8-615: US - Cost Per Unit & Supply of Cuvposa (US$), February’2026
Figure 8-616: US - BioThrax Orphan Drug Designation & Approval Year
Figure 8-617: US - Tinidazole Sulfate Orphan Drug Designation & Approval Year
Figure 8-618: US - Cost Per Unit & Supply of Tindamax (US$), February’2026
Figure 8-619: US - Ganciclovir Sulfate Orphan Drug Designation & Approval Year
Figure 8-620: US - Cost Per Unit & Supply of Zirgan (US$), February’2026
Figure 8-621: US - Hemangeol Sulfate Orphan Drug Designation & Approval Year
Figure 8-622: US - Cost Per Unit & Supply of Hemangeol (US$), February’2026
Figure 8-623: US - Acetadote Sulfate Orphan Drug Designation & Approval Year
Figure 8-624: US - Cost Per Unit & Supply of Acetadote (US$), February’2026
Figure 8-625: US - Tiglutik Sulfate Orphan Drug Designation & Approval Year
Figure 8-626: US - Cost Per Unit & Supply of Tiglutik (US$), February’2026
Figure 8-627: US - ATryn Sulfate Orphan Drug Designation & Approval Year
Figure 8-628: US - Tiglutik Sulfate Orphan Drug Designation & Approval Year
Figure 8-629: US - Caffeine Sulfate Orphan Drug Designation & Approval Year
Figure 8-630: US - Cost Per Unit & Supply of Cafcit (US$), February’2026
Figure 8-631: US - Factor XIII Concentrate Orphan Drug Designation & Approval Year
Figure 8-632: US - Diastat Orphan Drug Designation & Approval Year
Figure 8-633: US - Histrelin Acetate Orphan Drug Designation & Approval Year
Figure 8-634: US - Cholic Acid Orphan Drug Designation & Approval Year
Figure 8-635: US - Cost Per Unit & Supply of Cholbam (US$), February’2026
Figure 8-636: US - Basiliximab Orphan Drug Designation & Approval Year
Figure 8-637: US - Cost of Supply of Simulect (US$), February’2026
Figure 8-638: US - Dronabinol Orphan Drug Designation & Approval Year
Figure 8-639: US - Cost Per Unit & Supply of Marinol (US$), February’2026
Figure 8-640: US - Biktarvy Orphan Drug Designation & Approval Year
Figure 8-641: US - Cost Per Unit & Supply of Biktarvy (US$), February’2026
Figure 8-642: US - Follitropin Alfa Orphan Drug Designation & Approval Year
Figure 8-643: US - Dacogen Orphan Drug Designation & Approval Year
Figure 8-644: US - Cost Per Unit & Supply of 2.5mg, 5mg &10mg Revlimid (US$), February’2026
Figure 8-645: US - Cost Per Unit & Supply of 15m, 20mg & 25mg Revlimid (US$), February’2026
Figure 8-646: US - Arikayce Orphan Drug Designation & Approval Year
Figure 8-647: US - Cost Per Unit & Supply of Arikayce (US$), February’2026
Figure 8-648: US - Vutrisiran Orphan Drug Designation & Approval Year
Figure 8-649: US - Cost Per Unit & Supply of Amvuttra (US$), February’2026
Figure 8-650: US - Emflaza Orphan Drug Designation & Approval Year
Figure 8-651: US - Cost Per Unit & Supply of Emflaza Oral Tablet (US$), February’2026
Figure 8-652: US - Cost Per Unit & Supply of Emflaza Oral Suspension (US$), February’2026
Figure 8-653: US - Gocovri Orphan Drug Designation & Approval Year
Figure 8-654: US - Cost Per Unit & Supply of Gocovri (US$), February’2026
Figure 8-655: US - Asparlas Orphan Drug Designation & Approval Year
Figure 8-656: US - Cost Per Unit & Supply of Asparlas (US$), February’2026
Figure 8-657: US - Coagadex Orphan Drug Designation & Approval Year
Figure 8-658: US - Ampyra Orphan Drug Designation & Approval Year
Figure 8-659: US - Cost Per Unit & Supply of Ampyra (US$), February’2026
Figure 8-660: US - Anavip Orphan Drug Designation & Approval Year
Figure 8-661: US - Difluprednate Orphan Drug Designation & Approval Year
Figure 8-662: US - Cost Per Unit & Supply of Emflaza Oral Suspension (US$), February’2026
Figure 8-663: US - Balsalazide Disodium Orphan Drug Designation & Approval Year
Figure 8-664: US - Cost Per Unit & Supply of Colazal (US$), February’2026
Figure 8-665: US - Anascorp Orphan Drug Designation & Approval Year
Figure 8-666: US - Nithiodote Orphan Drug Designation & Approval Year
Figure 8-667: US - Cost Per Unit & Supply of Nithiodote (US$), February’2026
Figure 8-668: US - BabyBIG Orphan Drug Designation & Approval Year
Figure 8-669: US - Anacaulase Orphan Drug Designation & Approval Year
Figure 8-670: US - Cost Per Unit & Supply of NexoBrid (US$), February’2026
Figure 8-671: US - Cost Per Unit & Supply of Sarclisa (US$), February’2026
Figure 8-672: US - Miplyffa Orphan Drug Designation & Approval Year
Figure 8-673: US - Cost Per Unit & Supply of Miplyffa (US$), February’2026
Figure 8-674: US - Concizumab Orphan Drug Designation & Approval Year
Figure 8-675: US - Cost Per Unit & Supply of Alhemo (US$), February’2026
Figure 8-676: US - Avalglucosidase Alfa Orphan Drug Designation & Approval Year
Figure 8-677: US - Alyftrek Orphan Drug Designation & Approval Year
Figure 8-678: US - Cost Per Unit & Supply of Alyftrek (US$), February’2026
Figure 8-679: US - Somatrogon Orphan Drug Designation & Approval Year
Figure 8-680: US - Cost Per Unit & Supply of Ngenla (US$), February’2026
Figure 8-681: US - Velmanase Alfa Orphan Drug Designation & Approval Year
Figure 8-682: US - Omaveloxolone Orphan Drug Designation & Approval Year
Figure 8-683: US - Cost Per Unit & Supply of Skyclarys (US$), February’2026
Figure 8-684: US - Rozanolixizumab Orphan Drug Designation & Approval Year
Figure 8-685: US - Cost Per Unit & Supply of Rystiggo (US$), February’2026
Figure 8-686: US - Vorasidenib Orphan Drug Designation & Approval Year
Figure 8-687: US - Cost Per Unit & Supply of Voranigo (US$), February’2026
Figure 8-688: US - Seladelpar Orphan Drug Designation & Approval Year
Figure 8-689: US - Cost Per Unit & Supply of Livdelzi (US$), February’2026
Figure 8-690: US - Vamorolone Orphan Drug Designation & Approval Year
Figure 8-691: US - Cost Per Unit & Supply of Agamree (US$), February’2026
Figure 8-692: US - Tepotinib Orphan Drug Designation & Approval Year
Figure 8-693: US - Cost Per Unit & Supply of Tepmetko (US$), February’2026
Figure 8-694: US - Motixafortide Orphan Drug Designation & Approval Year
Figure 8-695: US - Acoramidis Orphan Drug Designation & Approval Year
Figure 8-696: US - Cost Per Unit & Supply of Attruby (US$), February’2026
Figure 8-697: US - Danicopan Orphan Drug Designation & Approval Year
Figure 8-698: US - Cost Per Unit & Supply of Voydeya (US$), February’2026
Figure 8-699: US - Levacetylleucine Orphan Drug Designation & Approval Year
Figure 8-700: US - Cost Per Unit & Supply of Aqneursa (US$), February’2026
Figure 8-701: US - Pafolacianine Orphan Drug Designation & Approval Year
Figure 8-702: US - Nedosiran Orphan Drug Designation & Approval Year
Figure 8-703: US - Cost Per Unit & Supply of Rivfloza (US$), February’2026
Figure 8-704: US - Treosulfan Orphan Drug Designation & Approval Year
Figure 8-705: US - Cost of Supply of Grafapex (US$), February’2026
Figure 8-706: US - Palovarotene Orphan Drug Designation & Approval Year
Figure 8-707: US - Cost Per Unit & Supply of Sohonos (US$), February’2026
Figure 8-708: US - Zolbetuximab Orphan Drug Designation & Approval Year
Figure 8-709: US - Cost of Supply of Vyloy (US$), February’2026
Figure 8-710: US - Imetelstat Orphan Drug Designation & Approval Year
Figure 8-711: US - Cost of Supply of Rytelo (US$), February’2026
Figure 8-712: US - Repotrectinib Orphan Drug Designation & Approval Year
Figure 8-713: US - Cost Per Unit & Supply of 40mg Augtyro (US$), February’2026
Figure 8-714: US - Cost Per Unit & Supply of 60mg Augtyro (US$), February’2026
Figure 8-715: US - Tofersen Orphan Drug Designation & Approval Year
Figure 8-716: US - Cost Per Unit & Supply of Qalsody (US$), February’2026
Figure 8-717: US - Sotatercept Orphan Drug Designation & Approval Year
Figure 8-718: US - Cost of Supply of Winrevair (US$), February’2026
Figure 8-719: US - Talquetamab Orphan Drug Designation & Approval Year
Figure 8-720: US - Cost Per Unit & Supply of Talvey (US$), February’2026
Figure 8-721: US - Rezafungin Orphan Drug Designation & Approval Year
Figure 8-722: US - Olezarsen Orphan Drug Designation & Approval Year
Figure 8-723: US - Cost Per Unit & Supply of Tryngolza (US$), February’2026
Figure 8-724: US - Penpulimab Orphan Drug Designation & Approval Year
Figure 8-725: US - Tislelizumab Orphan Drug Designation & Approval Year
Figure 8-726: US - Cost Per Unit & Supply of Tevimbra (US$), February’2026
Figure 8-727: US - Givinostat Orphan Drug Designation & Approval Year
Figure 8-728: US - Cost Per Unit & Supply of Duvyzat (US$), February’2026
Figure 8-729: US - Fexinidazole Orphan Drug Designation & Approval Year
Figure 8-730: US - Birch Triterpenes Orphan Drug Designation & Approval Year
Figure 8-731: US - Cost Per Unit & Supply of Filsuvez (US$), February’2026
Figure 8-732: US - Marstacimab Orphan Drug Designation & Approval Year
Figure 8-733: US - Adagrasib Orphan Drug Designation & Approval Year
Figure 8-734: US - Cost Per Unit & Supply of Krazati (US$), February’2026
Figure 8-735: US - Futibatinib Orphan Drug Designation & Approval Year
Figure 8-736: US - Cost Per Unit & Supply of Lytgobi (US$), February’2026
Figure 8-737: US - Eplontersen Orphan Drug Designation & Approval Year
Figure 8-738: US - Cost Per Unit & Supply of Wainua (US$), February’2026
Figure 8-739: US - Zanidatamab Orphan Drug Designation & Approval Year
Figure 8-740: US - Cost Per Unit & Supply of Ziihera (US$), February’2026
Figure 8-741: US - Zilucoplan Orphan Drug Designation & Approval Year
Figure 8-742: US - Cost Per Unit & Supply of Zilbrysq (US$), February’2026
Figure 8-743: US - Cost Per Unit & Supply of Loqtorzi (US$), February’2026
Figure 8-744: US - Elafibranor Orphan Drug Designation & Approval Year
Figure 8-745: US - Cost Per Unit & Supply of Iqirvo (US$), February’2026
Figure 8-746: US - Axatilimab Orphan Drug Designation & Approval Year
Figure 8-747: US - Cost Per Unit & Supply of Niktimvo (US$), February’2026
Figure 8-748: US - Retifanlimab Orphan Drug Designation & Approval Year
Figure 8-749: US - Cost Per Unit & Supply of Zynyz (US$), February’2026
Figure 8-750: US - Zenocutuzumab Orphan Drug Designation & Approval Year
Figure 8-751: US - Cost Per Unit & Supply of Bizengri (US$), February’2026
Figure 8-752: US - Elranatamab Orphan Drug Designation & Approval Year
Figure 8-753: US - Cost Per Unit & Supply of Elrexfio (US$), February’2026
Figure 8-754: US - Crinecerfont Orphan Drug Designation & Approval Year
Figure 8-755: US - Cost Per Unit & Supply of Crenessity Oral Capsules (US$), February’2026
Figure 8-756: US - Cost Per Unit & Supply of Crenessity Oral Solution (US$), February’2026
Figure 8-757: US - Revumenib Orphan Drug Designation & Approval Year
Figure 8-758: US - Cost Per Unit & Supply of Revuforj (US$), February’2026
Figure 8-759: US - Iptacopan Orphan Drug Designation & Approval Year
Figure 8-760: US - Cost Per Unit & Supply of Fabhalta (US$), February’2026
Figure 8-761: US - Asciminib Orphan Drug Designation & Approval Year
Figure 8-762: US - Cost Per Unit & Supply of Scemblix (US$), February’2026
Figure 8-763: US - Tarlatamab Orphan Drug Designation & Approval Year
Figure 8-764: US - Cost of Supply of Imdelltra (US$), February’2026
Figure 8-765: US - Teclistamab Orphan Drug Designation & Approval Year
Figure 8-766: US - Cost Per Unit & Supply of Tecvayli (US$), February’2026
Figure 8-767: US - Quizartinib Orphan Drug Designation & Approval Year
Figure 8-768: US - Cost Per Unit & Supply of Vanflyta (US$), February’2026
Figure 8-769: US - Palopegteriparatide Orphan Drug Designation & Approval Year
Figure 8-770: US - Tremelimumab Orphan Drug Designation & Approval Year
Figure 8-771: US - Cost Per Unit & Supply of Imjudo (US$), February’2026
Figure 8-772: US - Mavorixafor Orphan Drug Designation & Approval Year
Figure 8-773: US - Cost Per Unit & Supply of Xolremdi (US$), February’2026
Figure 8-774: US - Belzutifan Orphan Drug Designation & Approval Year
Figure 8-775: US - Cost Per Unit & Supply of Welireg (US$), February’2026
Figure 8-776: US - Pozelimab Orphan Drug Designation & Approval Year
Figure 8-777: US - Cost Per Unit & Supply of Veopoz (US$), February’2026
Figure 8-778: US - Tovorafenib Orphan Drug Designation & Approval Year
Figure 8-779: US - Cost Per Unit & Supply of Ojemda Oral Suspension (US$), February’2026
Figure 8-780: US - Cost Per Unit & Supply of Ojemda Oral Tablets (US$), February’2026
Figure 8-781: US - Momelotinib Orphan Drug Designation & Approval Year
Figure 8-782: US - Cost Per Unit & Supply of Ojjaara (US$), February’2026
Figure 8-783: US - Crovalimab Orphan Drug Designation & Approval Year
Figure 8-784: US - Cost Per Unit & Supply of Piasky (US$), February’2026
Figure 8-785: US - Belumosudil Orphan Drug Designation & Approval Year
Figure 8-786: US - Cost Per Unit & Supply of Rezurock (US$), February’2026
Figure 8-787: US - Cipaglucosidase Alfa Orphan Drug Designation & Approval Year
Figure 8-788: US - Cost Per Unit & Supply of Pombiliti (US$), February’2026
Figure 8-789: Pombiliti - Dosing Timeline
Figure 8-790: US - Nirogacestat Orphan Drug Designation & Approval Year
Figure 8-791: US - Cost Per Unit & Supply of Ogsiveo (US$), February’2026
Figure 8-792: US - Fitusiran Orphan Drug Designation & Approval Year
Figure 8-793: US - Cost Per Unit & Supply of Qfitlia (US$), February’2026
Figure 8-794: US - Prademagene Zamikeracel Orphan Drug Designation & Approval Year
Figure 9-1: Amvuttra - Global Sales (US$ Million), 2022-2025
Figure 9-2: Amvuttra - Global Sales by Region (US$ Million), 2022-2025
Figure 9-3: Krazati - Global Sales (US$ Million), 2023-2025
Figure 9-4: Krazati - Global Sales by Region (US$ Million), 2023-2025
Figure 9-5: Tecvayli - Global Sales (US$ Million), 2022-2025
Figure 9-6: Tecvayli - Global Sales by Region (US$ Million), 2022-2025
Figure 9-7: Imjudo - Global Sales (US$ Million), 2024-2025
Figure 9-8: Imjudo - Global Sales by Region (US$ Million), 2024-2025
Figure 9-9: Rezurock - Global Sales (US$ Million), 2022-2025
Figure 9-10: Rezurock - Global Sales by Region (US$ Million), 2022-2025
Figure 9-11: Elrexfio - Global Sales (US$ Million), 2024-2025
Figure 9-12: Elrexfio - Global Sales by Region (US$ Million), 2024-2025
Figure 9-13: Fabhalta - Global Sales (US$ Million), 2023-2025
Figure 9-14: Filspari - Global Sales (US$ Million), 2023-2025
Figure 9-15: Wainua - Global Sales (US$ Million), 2024-2025
Figure 9-16: Wainua - Global Sales by Region (US$ Million), 2024-2025
Figure 9-17: Jaypirca - Global Sales (US$ Million), 2024-2025
Figure 9-18: Piasky - Global Sales (US$ Million), 2024-2025
Figure 9-19: Piasky - Global Sales by Region (US$ Million), 2024-2025
Figure 9-20: Imdelltra - Global Sales (US$ Million), 2024-2025
Figure 9-21: Imdelltra - Global Sales by Region (US$ Million), 2024-2025
Figure 9-22: Skyclarys - Global Sales (US$ Million), 2023-2025
Figure 9-23: Skyclarys - Global Sales by Region (US$ Million), 2023-2025
Figure 9-24: Qalsody - Global Sales (US$ Million), 2023-2025
Figure 9-25: Qalsody - Global Sales by Region (US$ Million), 2023-2025
Figure 9-26: Talvey - Global Sales (US$ Million), 2023-2025
Figure 9-27: Talvey - Global Sales by Region (US$ Million), 2023-2025
Figure 9-28: Iqirvo - Global Sales (US$ Million), 2023-2025
Figure 9-29: Iqirvo - Global Sales by Region (US$ Million), 2023-2025
Figure 9-30: Xen[pozyme - Global Sales (US$ Million), 2023-2025
Figure 9-31: Xenpozyme - Global Sales by Region (US$ Million), 2023-2025
Figure 9-32: Sohonos - Global Sales (US$ Million), 2023-2025
Figure 9-33: Sohonos - Global Sales by Region (US$ Million), 2023-2025
Figure 9-34: Bylvay - Global Sales (US$ Million), 2023-2025
Figure 9-35: Bylvay - Global Sales by Region (US$ Million), 2023-2025
Figure 9-36: Zynyz - Global Sales (US$ Million), 2023-2025
Figure 9-37: Ojjaara - Global Sales (US$ Million), 2023-2025
Figure 9-38: Ojjaara - Global Sales by Region (US$ Million), 2023-2025
Figure 9-39: Attruby - Global Sales (US$ Million), 2024-2025
Figure 9-40: Winrevair - Global Sales (US$ Million), 2024-2025
Figure 9-41: Winrevair - Global Sales by Region (US$ Million), 2024-2025
Figure 9-42: Niktimvo - Global Sales (US$ Million), 2025
Figure 9-43: Welireg - Global Sales (US$ Million), 2022-2025
Figure 9-44: Scemblix - Global Sales (US$ Million), 2022-2025
Figure 9-45: Tryngolza - Global Sales (US$ Million), 2025
Figure 9-46: Yorvipath - Global Sales (US$ Million), 2024-2025
Figure 9-47: Xolremdi - Global Sales (US$ Million), 2024-2025
Figure 9-48: Livtencity - Global Sales (US$ Million), 2021-2025
Figure 9-49: Livtencity - Global Sales by Region (US$ Million), 2021-2025
Figure 9-50: Zynlonta - Global Sales (US$ Million), 2021-2025
Figure 9-51: Lytgobi - Global Sales (US$ Million), 2023-2025
Figure 9-52: NexoBrid - Global Sales (US$ Million), 2021-2025
Figure 9-53: Pombiliti & Opfolda - Global Sales (US$ Million), 2022-2025
Figure 9-54: Vanflyta - Global Sales (US$ Million), 2023-2025
Figure 9-55: Alyftrek - Global Sales (US$ Million), 2025
Figure 9-56: Tafinlar - Global Sales (US$ Million), 2019-2025
Figure 9-57: Tafinlar - Global Sales by Region (US$ Million), Q1’2025
Figure 9-58: Tafinlar - Global Sales by Region (US$ Million), 2024
Figure 9-59: Kimmtrak- Global Sales (US$ Million), 2022-2025
Figure 9-60: Kimmtrak- Global Sales by Region (US$ Million), 2023-2025
Figure 9-61: Rezlidhia- Global Sales (US$ Million), 2022-2025
Figure 9-62: Agamree - Global Sales (US$ Million), 2024-2025
Figure 9-63: Daybue - Global Sales (US$ Million), 2023-2025
Figure 9-64: Ogsiveo - Global Sales (US$ Million), 2023-2025
Figure 9-65: Joenja - Global Sales (US$ Million), 2023-2025
Figure 9-66: Joenja - Global Sales by Region (US$ Million), 2023-2025
Figure 9-67: Terlivaz - Global Sales (US$ Million), 2022-2025
Figure 9-68: Crenessity - Global Sales (US$ Million), 2024-2025
Figure 9-69: Revuforj - Global Sales (US$ Million), 2024-2025
Figure 9-70: Ojemda - Global Sales (US$ Million), 2024-2025
Figure 9-71: Rytelo - Global Sales (US$ Million), 2024-2025
Figure 9-72: Ziihera - US Sales (US$ Million), 2024-2025
Figure 9-73: Empaveli - Global Sales (US$ Million), 2021-2025
Figure 9-74: Augtyro - Global Sales (US$ Million), 2023-2024
Figure 9-75: Augtyro - Global Sales by Region (US$ Million), 2024
Figure 9-76: Miplyffa - Global Sales (US$ Million), 2024-2025
Figure 9-77: Vonjo - Global Sales (US$ Million), 2023-2025
Figure 9-78: Vyloy - Global Sales (US$ Million), 2024-2025
Figure 9-79: Joenja - Global Sales by Region (US$ Million), 2023-2025
Figure 9-80: Enjaymo - Global Sales (US$ Million), 2022-2025
Figure 9-81: Aphexda - Global Sales (US$ Million), 2022-2025
Figure 9-82: Loqtorzi - US Sales (US$ Million), 2022-2025
Figure 9-83: Ztalmy - Global Sales (US$ Million), 2022-2025
Figure 9-84: Zilbrysq - Global Sales (US$ Million), 2024-2025
Figure 9-85: Zilbrysq - Global Sales by Region (US$ Million), 2024-2025
Figure 9-86: Rystiggo - Global Sales (US$ Million), 2023-2025
Figure 9-87: Rystiggo - Global Sales by Region (US$ Million), 2023-2025
Figure 9-88: Tevimbra - Global Sales (US$ Million), 2024-2025
Figure 9-89: Zolgensma - Global Sales (US$ Million), 2021-2025
Figure 9-90: Zolgensma - Global Sales by Region (US$ Million), 2025
Figure 9-91: Zolgensma - US Sales (US$ Million), 2021-2025
Figure 9-92: Zolgensma - ROW Sales (US$ Million), 2021-2025
Figure 9-93: Lynparza - Global Sales (US$ Million), 2021-2025
Figure 9-94: Lynparza - Global Sales by Region (US$ Million), 2025
Figure 9-95: Lynparza - US Sales (US$ Million), 2021-2025
Figure 9-96: Lynparza - Europe Sales (US$ Million), 2021-2025
Figure 9-97: Lynparza - Emerging Markets Sales (US$ Million), 2021-2025
Figure 9-98: Lynparza - ROW Sales (US$ Million), 2021-2025
Figure 9-99: Nplate - Global Sales (US$ Million), 2021-2025
Figure 9-100: Nplate - Global Sales by Region (US$ Million), 2022-2025
9 101: Lumakras - Global Sales (US$ Million), 2021-2025
Figure 9-102: Lumakras - US Sales (US$ Million), 2021-2025
Figure 9-103: Lumakras - ROW Sales (US$ Million), 2021-2025
Figure 9-104: Repatha - Global Sales (US$ Million), 2021-2025
Figure 9-105: Repatha - Global Sales by Region (US$ Million), 2021-2025
Figure 9-106: Otezla - Global Sales (US$ Million), 2021-2025
Figure 9-107: Otezla - Global Sales by Region (US$ Million), 2021-2025
Figure 9-108: Kyprolis - Global Sales (US$ Million), 2021-2025
Figure 9-109: Kyprolis - Global Sales by Region (US$ Million), 2021-2025
Figure 9-110: Opdivo - Global Sales (US$ Million), 2021-2025
Figure 9-111: Opdivo - Global Sales by Region (US$ Million), 2025
Figure 9-112: Opdualag - Global Sales (US$ Million), 2022-2025
Figure 9-113: Opdualag - Global Sales by Region (US$ Million), 2022-2025
Figure 9-114: Braftovi + Mektovi - Global Sales (US$ Million), 2021-2025
Figure 9-115: Braftovi + Mektovi - Global Sales by Region (US$ Million), 2022-2025
Figure 9-116: Lorbrena - Global Sales (US$ Million), 2021-2025
Figure 9-117: Lorbrena - Global Sales by Region (US$ Million), 2021-2025
Figure 9-118: Besponsa - Global Sales (US$ Million), 2021-2023
Figure 9-119: Besponsa - Global Sales (US$ Million), Q1-Q4’2022
Figure 9-120: Besponsa - US Sales (US$ Million), 2021-2023
Figure 9-121: Besponsa - US Sales (US$ Million), Q1-Q4’2022
Figure 9-122: Besponsa - Global Sales by Region (US$ Million), 2022
Figure 9-123: Besponsa - ROW Sales (US$ Million), 2021-2023
Figure 9-124: Actemra - Global Sales (US$ Million), 2021-2025
Figure 9-125: Actemra - Global Sales by Region (US$ Million), 2025
Figure 9-126: Actemra - US Sales (US$ Million), 2021-2025
Figure 9-127: Actemra - Europe Sales (US$ Million), 2021-2025
Figure 9-128: Actemra - Japan Sales (US$ Million), 2021-2025
Figure 9-129: Actemra - ROW Sales (US$ Million), 2021-2025
Figure 9-130: Tecentriq - Global Sales (US$ Million), 2021-2025
Figure 9-131: Tecentriq - Global Sales by Region (US$ Million), 2025
Figure 9-132: Tecentriq - US Sales (US$ Million), 2021-2025
Figure 9-133: Tecentriq - Europe Sales (US$ Million), 2021-2025
Figure 9-134: Tecentriq - Japan Sales (US$ Million), 2021-2025
Figure 9-135: Tecentriq - ROW Sales (US$ Million), 2021-2025
Figure 9-136: Polivy - Global Sales (US$ Million), 2021-2025
Figure 9-137: Polivy - Global Sales by Region (US$ Million), 2025
Figure 9-138: Polivy - US Sales (US$ Million), 2021-2025
Figure 9-139: Polivy - Europe Sales (US$ Million), 2021-2025
Figure 9-140: Polivy - Japan Sales (US$ Million), 2021-2025
Figure 9-141: Polivy - ROW Sales (US$ Million), 2021-2025
Figure 9-142: Luxturna - Global Sales (US$ Million), 2023-2025
Figure 9-143: Enspryng - Global Sales (US$ Million), 2021-2025
Figure 9-144: Enspryng - Global Sales by Region (US$ Million), 2025
Figure 9-145: Enspryng - US Sales (US$ Million), 2021-2025
Figure 9-146: Enspryng - Europe Sales (US$ Million), 2021-2025
Figure 9-147: Enspryng - Japan Sales (US$ Million), 2021-2025
Figure 9-148: Enspryng - ROW Sales (US$ Million), 2021-2025
Figure 9-149: Evrysdi - Global Sales (US$ Million), 2021-2025
Figure 9-150: Evrysdi - Global Sales by Region (US$ Million), 2025
Figure 9-151: Evrysdi - US Sales (US$ Million), 2020-2025
Figure 9-152: Evrysdi - Europe Sales (US$ Million), 2021-2025
Figure 9-153: Evrysdi - Japan Sales (US$ Million), 2021-2025
Figure 9-154: Evrysdi - ROW Sales (US$ Million), 2021-2025
Figure 9-155: Trikafta - Global Sales (US$ Million), 2019-2025
Figure 9-156: Trikafta - Global Sales (US$ Million), Q1-Q4’2024
Figure 9-157: Kalydeco - Global Sales (US$ Million), 2019-2023
Figure 9-158: Symdeko - Global Sales (US$ Million), 2019-2023
Figure 9-159: Orkambi - Global Sales (US$ Million), 2019-2023
Figure 9-160: Reblozyl - Global Sales (US$ Million), 2020-2025
Figure 9-161: Reblozyl - Global Sales by Region (US$ Million), 2020-2025
Figure 9-162: Yervoy - Global Sales (US$ Million), 2021-2025
Figure 9-163: Yervoy - Global Sales by Region (US$ Million), 2021-2025
Figure 9-164: Eylea - Global Sales (US$ Million), 2021-2025
Figure 9-165: Eylea - Global Sales by Region (US$ Million), 2021-2025
Figure 9-166: Evkeeza - US Sales (US$ Million), 2021-2025
Figure 9 167: Skytrofa - Global Sales (US$ Million), 2021-2025
Figure 9-168: Elahere - Global Sales (US$ Million), 2022-2025
Figure 9-169: Elahere - Global Sales by Region (US$ Million), 2022-2025
Figure 9-170: Jakafi - Global Sales (US$ Million), 2021-2025
Figure 9-171: Jakafi - Global Sales by Region (US$ Million), 2021-2025
Figure 9-172: Ninlaro - Global Sales (US$ Million), 2022-2025
Figure 9-173: Ninlaro - Global Sales by Region (US$ Million), 2022-2025
Figure 9-174: Takhzyro - Global Sales (US$ Million), 2022-2025
Figure 9-175: Takhzyro - 2022-2025 (US$ Million), 2024-2025
Figure 9-176: Adcetris - US Sales (US$ Million), 2020-2025
Figure 9-177: Onpattro - Global Sales (US$ Million), 2021-2025
Figure 9-178: Onpattro - Global Sales by Region (US$ Million), 2025
Figure 9-179: Onpattro - Global Sales by Region (US$ Million), 2021-2025
Figure 9-180: Orladeyo - Global Sales (US$ Million), 2021-2025
Figure 9-181: Orladeyo - Global Sales by Region (US$ Million), 2021-2025
Figure 9-182: Livmarli - Global Sales (US$ Million), 2021-2025
Figure 9-183: Bendeka - Global Sales (US$ Million), 2021-2025
Figure 9-184: Firdapse - Global Sales (US$ Million), 2021-2025
Figure 9-185: Vyvgart - Global Sales (US$ Billion), 2022-2025
Figure 9-186: Tagrisso - Global Sales (US$ Million), 2021-2025
Figure 9-187: Tagrisso - Global Sales by Region (US$ Million), 2025
Figure 9-188: Tagrisso - US Sales (US$ Million), 2021-2025
Figure 9-189: Tagrisso - Europe Sales (US$ Million), 2021-2025
Figure 9-190: Tagrisso - Emerging Markets Sales (US$ Million), 2021-2025
Figure 9-191: Tagrisso - ROW Sales (US$ Million), 2021-2025
Figure 9-192: Koselugo - Global Sales (US$ Million), 2021-2025
Figure 9-193: Koselugo - Global Sales by Region (US$ Million), 2025
Figure 9-194: Koselugo - US Sales (US$ Million), 2021-2025
Figure 9-195: Koselugo - Europe Sales (US$ Million), 2021-2025
Figure 9-196: Koselugo - Emerging Markets Sales (US$ Million), 2021-2025
Figure 9-197: Koselugo - ROW Sales (US$ Million), 2023-2025
Figure 9-198: Enhertu - Global Sales (US$ Million), 2021-2025
Figure 9-199: Voxzogo - Global Sales (US$ Million), 2020-2025
Figure 9-200: Palynziq - Global Sales (US$ Million), 2019-2025
Figure 9-201: Ravicti - Global Sales (US$ Million), 2021-2025
Figure 9-202: Tecartus - Global Sales (US$ Million), 2021-2025
Figure 9-203: Tecartus - Global Sales by Region (US$ Million), 2025
Figure 9-204: Tecartus - US Sales (US$ Million), 2021-2025
Figure 9-205: Tecartus - Europe Sales (US$ Million), 2021-2025
Figure 9-206: Tecartus - ROW Sales (US$ Million), 2021-2025
Figure 9-207: Darzalex - Global Sales (US$ Million), 2021-2025
Figure 9-208: Darzalex - Global Sales by Region (US$ Million), 2021-2025
Figure 9-209: Opsumit - Global Sales (US$ Million), 2021-2025
Figure 9-210: Opsumit - Global Sales by Region (US$ Million), 2021-2025
Figure 9-211: Uptravi - Global Sales (US$ Million), 2021-2025
Figure 9-212: Uptravi - Global Sales by Region (US$ Million), 2021-2025
Figure 9-213: Cyramza - Global Sales (US$ Million), 2021-2025
Figure 9-214: Cyramza - Global Sales by Region (US$ Million), 2021-2025
Figure 9-215: Alimta - Global Sales (US$ Million), 2021-2025
Figure 9-216: Alimta - Global Sales by Region (US$ Million), 2021-2025
Figure 9-217: Tepezza - Global Sales (US$ Million), 2020-2025
Figure 9-218: Tepezza - Global Sales by Region (US$ Million), 2025
Figure 9-219: Uplizna - Global Sales (US$ Million), 2020-2025
Figure 9-220: Uplizna - Global Sales by Region (US$ Million), 2023-2025
Figure 9-221: Spinraza - Global Sales (US$ Million), 2021-2025
Figure 9-222: Spinraza - Global Sales by Region (US$ Million), 2021-2025
Figure 9-223: Epidiolex - Global Sales (US$ Million), 2021-2025
Figure 9-224: Xywav - Global Sales (US$ Million), 2021 - 2025
Figure 9-225: Xyrem - Global Sales (US$ Billion), 2021-2025
Figure 9-226: Vyxeos - Global Sales (US$ Million), 2019-2025
Figure 9-227: Rylaze - Global Sales (US$ Million), 2021-2025
Figure 9-228: Pyrukynd - Global Sales (US$ Million), 2022-2025
Figure 9-229: Hemlibra - Global Sales (US$ Million), 2021-2025
Figure 9-230: Hemlibra - Global Sales by Region (US$ Million), 2025
Figure 9-231: Hemlibra - US Sales (US$ Million), 2021-2025
Figure 9-232: Hemlibra - Europe Sales (US$ Million), 2021-2025
Figure 9-233: Hemlibra - Japan Sales (US$ Million), 2021-2025
Figure 9-234: Hemlibra - ROW Sales (US$ Million), 2021-2025
Figure 9-235: Prolia - Global Sales (US$ Million), 2021-2025
Figure 9-236: Prolia - - Global Sales by Region (US$ Million), 2021-2025
Figure 9-237: Venclexta - Global Sales (US$ Million), 2021-2025
Figure 9-238: Venclexta - Global Sales by Region (US$ Million), Q1’2025
Figure 9-239: Venclexta - US Sales (US$ Million), 2021-2025
Figure 9-240: Xgeva - Global Sales (US$ Million), 2021-2025
Figure 9-241: Xgeva - Global Sales by Region (US$ Million), 2021-2025
Figure 9-242: Tavneos - Global Sales (US$ Million), 2022-2025
Figure 9-243: Tavneos - Global Sales by Region (US$ Million), 2022-2025
Figure 9-244: Cometriq - US Sales (US$ Million), 2021-2025
Figure 9-245: Cabometyx - US Sales (US$ Million), 2021-2025
Figure 9-246: Carvykti - Global Sales (US$ Million), 2022-2025
Figure 9-247: Carvykti - Global Sales by Region (US$ Million), 2022-2025
Figure 9-248: Kymriah - Global Sales (US$ Million), 2019-2025
Figure 9-249: Pomalyst - Global Sales (US$ Million), 2021-2025
Figure 9-250: Pomalyst - Global Sales by Region (US$ Million), 2021-2025
Figure 9-251: Xiaflex - Global Sales (US$ Million), 2021-2025
Figure 9-252: Recorlev - Global Sales (US$ Million), 2022-2025
Figure 9-253: Gleevec - Global Sales (US$ Million), 2019-2023
Figure 9-254: Gleevec - Global Sales (US$ Million), Q1-Q4’ 2022
Figure 9-255: Turalio - Global Sales (US$ Million), 2023-2025
Figure 9-256: Bosulif - Global Sales (US$ Million), 2021-2025
Figure 9-257: Bosulif - Global Sales by Region (US$ Million), 2019-2025
Figure 9-258: Brineura - Global Sales (US$ Million), 2021-2025
Figure 9-259: Botox Therapeutic - Global Sales (US$ Million), 2021-2025
Figure 9-260: Botox Therapeutic - Global Sales by Region (US$ Million), 2021-2025
Figure 9-261: Humira - Global Sales (US$ Million), 2021-2025
Figure 9-262: Humira - Global Sales by Region (US$ Million), 2021-2025
Figure 9-263: Poteligeo - Global Sales (US$ Million), 2023-2025
Figure 9-264: Crysvita - Global Sales (US$ Million), 2023-2025
Figure 9-265: Promacta - Global Sales (US$ Million), 2021-2025
Figure 9-266: Promacta - Global Sales by Region (US$ Million), 2021-2025
Figure 9-267: Eloctate - Global Sales (US$ Million), 2023-2025
Figure 9-268: Eloctate - Global Sales by Region (US$ Million), 2023-2025
Figure 9-269: Xospata - Global Sales (US$ Million), 2023-2025
Figure 9-270: Xospata - Global Sales by Region (US$ Million), 2023-2025
Figure 9-271: Defitelio - Global Sales (US$ Million), 2021-2025
Figure 9-272: Praluent - Global Sales (US$ Million), 2021-2025
Figure 9-273: Praluent - Global Sales by Region (US$ Million), 2021-2025
Figure 9-274: Mozobil - Global Sales (US$ Million), 2021-2025
Figure 9-275: Mozobil - Global Sales by Region (US$ Million), 2021-2025
Figure 9-276: Yescarta - Global Sales (US$ Million), 2021-2025
Figure 9-277: Yescarta - Global Sales by Region (US$ Million), 2021-2025
Figure 9-278: Lunsumio - Global Sales (US$ Million), 2022-2025
Figure 9-279: Lunsumio - Global Sales by Region (US$ Million), 2025
Figure 9-280: Lunsumio - US Sales (US$ Million), 2022-2025
Figure 9-281: Lunsumio - Europe Sales (US$ Million), 2022-2025
Figure 9-282: Lunsumio - Japan Sales (US$ Million), 2022-2025
Figure 9-283: Lunsumio - ROW Sales (US$ Million), 2022-2025
Figure 9-284: Keytruda - Global Sales (US$ Million), 2021-2025
Figure 9-285: Keytruda - Global Sales by Region (US$ Million), 2021-2025
Figure 9-286: Pemazyre - Global Sales (US$ Million), 2021-2025
Figure 9-287: Camzyos - Global Sales (US$ Million), 2022-2025
Figure 9-288: Camzyos - Global Sales by Region (US$ Million), 2022-2025

List of Tables

Table 2-1: US - FDA Categorization for Disease or Condition
Table 2-2: US - FDA Criteria for Orphan & non-Orphan Drug Approvals
Table 8-1: Tafinlar - Recommended Weight-Adjusted Dose
Table 8-2: Ocaliva - Dosage Regimen by Disease Stage
Table 8-3: Nivolumab - FDA Orphan Drug Designation & Approvals
Table 8-4: Opdivo - Recommended Dosages for Monotherapy
Table 8-5: Opdivo - Recommended Dosages in Combination with Other Therapeutic Agents
Table 8-6: 9.5 mg/mL Livmarli Oral Solution - Volume Per Dose (mL) by Weight for Patients With ALGS
Table 8-7: Livmarli Tablets - Dosage by Weight for Patients With ALGS
Table 8-8: 19mg/mL Livmarli Oral Solution - Volume Per Dose (mL) by Weight for Patients With PFIC
Table 8-9: Livmarli Tablets - Dosage by Weight for Patients With ALGS
Table 8-10: Firdapse - Recommended Oral Dosage for the Treatment of LEMS in Adults & Pediatric Patients 6 Years of Age & Older
Table 8-11: Evrysdi - Adult & Pediatric Dosing Regimen by Age & Body Weight
Table 8-12: Hetlioz LQ - Recommended Dosage for the Treatment of Nighttime Sleep Disturbances in SMS in Pediatric Patients 3 Years to 15 Years of Age
Table 8-13: Ipilimumab - FDA Orphan Drug Designation & Approvals
Table 8-14: Yervoy - Recommended Dosages as a Single Agent
Table 8-15: Yervoy - Recommended Dosages in Combination with Other Therapeutic Agents
Table 8-16: Bevacizumab - FDA Orphan Drug Designations
Table 8-17: Tpoxx Capsules - Recommended Dosage & Preparation Instructions in Pediatric Patients Weighing at Least 13 kg & Adults
Table 8-18: Koselugo Capsules - Recommended Dosage Based on BSA
Table 8-19: Koselugo Oral Granule- Recommended Dosage Based on BSA
Table 8-20: Actemra - Recommended Dosages for Polyarticular Juvenile Idiopathic Arthritis
Table 8-21: Actemra - Recommended Dosages for Cytokine Release Syndrome (CRS)
Table 8-22: Tecentriq - Recommended Dosage in Combination with Other Therapeutic Agents
Table 8-23: Kimmtrak - Recommended Dosage for Treatment of Unresectable or Metastatic Uveal Melanoma
Table 8-24: Jakafi - Starting Doses for Myelofibrosis
Table 8-25: Kalydeco - Recommended Dosage in Pediatric Patients Aged 1 Month to Less than 6 Years
Table 8-26: Symdeko - Recommended Dosage for Adult and Pediatric Patients aged 6 Years & Older
Table 8-27: Trikafta - Recommended Dosage for Adult & Pediatric Patients Aged 6 Years & Older
Table 8-28: Orkambi - Recommended Oral Dosage of in Patients Aged 1 Year and Older
Table 8-29: Voxzogo - Recommended Daily Dosage and Injection Volume
Table 8-30: Actimmune - Recommended Dosage for Treatment of Patients with CGD & SMO
Table 8-31: Lisocabtagene maraleucel - FDA Orphan Drug Designation & Approvals
Table 8-32: Tazemetostat - FDA Orphan Drug Designation & Approvals
Table 8-33: Otezla - Dosage Titration Schedule for Behçet’s Disease
Table 8-34: Otezla - Dosage Titration Schedule for Pediatric Patients 6 Years of Age and Older and Weighing at Least 20 kg with Behçet’s Disease
Table 8-35: Gammagard Liquid - Dosage and Administration for Multifocal Motor Neuropathy
Table 8-36: Fintepla - Recommended Titration Schedule
Table 8-37: Daybue - Recommended Dosage in Patients 2 Years of Age and Older
Table 8-38: Juxtapid - Recommended Regimen for Titrating Dosage
Table 8-39: Myalept - Recommended Dosage
Table 8-40: Empliciti - Recommended Dosing Schedule in Combination with Lenalidomide & Dexamethasone
Table 8-41: Empliciti - Recommended Dosing Schedule in Combination with Pomalidomide & Dexamethasone
Table 8-42: Kcentra - Dosage Required for Reversal of VKA Anticoagulation in Patients with acute major bleeding or need for an urgent surgery/invasive procedure
Table 8-43: Kyprolis - 20/70 mg/m2 Once Weekly (30-Minute Infusion)
Table 8-44: Kyprolis - 20/56 mg/m2 Twice Weekly (30-Minute Infusion)
Table 8-45: Sirturo - Recommended Dosage in Adult Patients
Table 8-46: Sirturo - Recommended Dosage of in Pediatric Patients
Table 8-47: Daratumumab - FDA Orphan Drug Designation & Approvals
Table 8-48: Darzalex - Dosing Schedule in Combination With Lenalidomide or Pomalidomide (4-Week Cycle) and Low-Dose Dexamethasone and for Monotherapy
Table 8-49: Darzalex - Dosing Schedule in Combination With Bortezomib, Melphalan and Prednisone ([VMP], 6-Week Cycle)
Table 8-50: Darzalex - Dosing Schedule in Combination With Bortezomib, Thalidomide and Dexamethasone ([VTd]; 4-Week Cycle)
Table 8-51: Darzalex - Dosing Schedule With Bortezomib and Dexamethasone (3-Week Cycle)
Table 8-52: Darzalex - Dosing Schedule With Carfilzomib and Dexamethasone (4-Week Cycle)
Table 8-53: Darzalex Faspro - Dosing Schedule in Combination With Bortezomib, Cyclophosphamide and Dexamethasone (4-week cycle)
Table 8-54: Endari - Recommended Dosing
Table 8-55: Aldesleukin - FDA Orphan Drug Designation & Approvals
Table 8-56: Coartem - Tablets Per Dose by Bodyweight in Pediatric Patients
Table 8-57: Nulibry - Recommended Dose
Table 8-58: Valtoco - Recommended Dosage for Adults and Pediatric Patients 2 Years of Age and Older
Table 8-59: Vistogard - Recommended Dose for Pediatric Patients
Table 8-60: Xuriden - Recommended Dosage by Body Weight
Table 8-61: Ascor - Recommended Dose & Infusion Rate of Diluted Solution
Table 8-62: BAT Dosing Guide and Intravenous Infusion Rate
Table 8-63: BAT - Pediatric Dosing Guide Based on Salisbury Rule (1 Year to < 17 Years)
Table 8-64: Priftin - Weight Based Dose for Treatment of Latent Tuberculosis Infection in Children
Table 8-65: Gralise - Recommended Titration Schedule
Table 8-66: Pedmark - Recommended Dose
Table 8-67: Palynziq - Recommended Dosing Regimen
Table 8-68: Tembexa - Recommended Dosage
Table 8-69: Rituximab - Orphan Drug Designation & Approvals
Table 8-70: Rituxan - Recommended Dosage
Table 8-71: Cresemba - Recommended Dosage Regimen in Adult Patients
Table 8-72: Cresemba - Recommended Dosage Regimen in Adult Patients
Table 8-73: Oxlumo - Weight-Based Dosing Regimen
Table 8-74: Velcade - Dosage Regimen for Patients with Previously Untreated Multiple Myeloma
Table 8-75: Xyrem - Recommended Adult Dose Regimen
Table 8-76: Xyrem - Recommended Pediatric Dosage for Patients 7 Years of Age and Older
Table 8-77: Vyxeos - Dose & Schedule
Table 8-78: Pyrukynd - Dose Titration Schedule
Table 8-79: Brentuximab Vedotin - FDA Orphan Drug Designation & Approvals
Table 8-80: Adcetris - Recommended Dosage
Table 8-81: Rebinyn - Dosing for on-demand Treatment and Control of Bleeding Episodes
Table 8-82: Rebinyn - Dosing for Perioperative Management
Table 8-83: Impavido - Recommended Dosage
Table 8-84: Imatinib - FDA Orphan Drug Designation & Approvals
Table 8-85: Eloctate - Dosing for Control of Bleeding Episodes
Table 8-86: Eloctate - Dosing for Perioperative Management
Table 8-87: Rozlytrek - Recommended Dosage for Adults and Pediatric patients for Treatment of NTRK Gene Fusion-Positive Solid Tumors
Table 8-88: Rozlytrek - Recommended Dosage for Pediatric Patients Older than 6 Months for the Treatment of NTRK Gene Fusion-Positive Solid Tumors
Table 8-89: Humira - US Orphan Drug Designations
Table 8-90: Humira - Recommended Dosage for Adolescent Patients 12 years of Age & Older Weighing at least 30 kg with Hidradenitis Suppurativa
Table 8-91: Humira - Recommended Dosage for Pediatric Patients 6 Years of Age & Older With Crohn’s Disease
Table 8-92: Humira - Recommended Dosage for Patients 2 Years of Age & Older With Polyarticular Juvenile Idiopathic Arthritis
Table 8-93: Humira - Recommended Dosage for Pediatric Patients 5 Years of Age & Older With Ulcerative Colitis
Table 8-94: Serostim - Body Weight-Based Dosage Recommendation
Table 8-95: Obizur - Dosage for on-Demand Management of Bleeding Episodes
Table 8-96: Mekinist - Recommended Dosage & Treatment Cost per Month (30 days) in Pediatric Patients (Weight-Based) (US$), February’2026
Table 8-97: Lunsumio - Recommended Dose, Schedule & Cost of Treatment (21-day Treatment Cycle) (US$), February’2026
Table 8-98: Bylvay - Recommended Dosage for PFIC in Patients aged 3 months and older (40 mcg/kg/day)
Table 8-99: Bylvay - Recommended Dosage for ALGS in Patients aged 12 months and older (120 mcg/kg/day)
Table 8-100: Ultomiris - IV Administration of Weight-Based Dosing Regimen - PNH, aHUS, gMG, or NMOSD
Table 8-101: Cuvrior - Recommended Starting Total Daily Dosage When Switching from Penicillamine
Table 8-102: HepaGam B - Dosing Regimen for HBV Related Liver Transplant Patients
Table 8-103: VariZIG - Recommended Dose & Volume
Table 8-104: CytoGam - Recommended Dosage According to Type of Transplant & Cost per Dose (US$), February’2026
Table 8-105: Astagraf XL - Recommended Starting Daily Dosage Regimen
Table 8-106: US - Tacrolimus Orphan Drug Designation & Approval Year
Table 8-107: Prograf Capsules - Summary of Initial Dosage Recommendations and Whole Blood Trough Concentration Range in Adults
Table 8-108: Prograf Capsule & Granules - Summary of Initial Dosage Recommendations and Whole Blood Trough Concentration Range in Children
Table 8-109: Ztalmy - Recommended Titration Schedule for Patients Weighing 28kg or Less
Table 8-110: Ztalmy - Recommended Titration Schedule for Patients Weighing More Than 28kg
Table 8-111: US - Keytruda Orphan Drug Designation Year
Table 8-112: US - Keytruda Orphan Drug Designation Year
Table 8-113: Venclexta - Recommended Dosing Schedule for Ramp-up Phase for Patients with CLL
Table 8-114: Venclexta - Recommended Dosing Schedule for RamUp Phase with Cost per Dose for AML (US$), June’2023
Table 8-115: Corlanor - Dose Adjustments for Adults
Table 8-116: Esbriet - Dosage Titration in Patients with IPF
Table 8-117: Idelvion - Dosing for on-Demand Treatment & Control of Bleeding Episodes
Table 8-118: Idelvion -Dosing for Perioperative Management of Bleeding, February’2026
Table 8-119: Mepsevii - Recommended Administration Infusion Rate Schedule by Patient Weight at 4mg/kg
Table 8-120: Cystagon - Recommended Maintenance Dose
Table 8-121: Sabril - Infant Dosing Table
Table 8-122: Vonvendi - Dosing Guidelines for Treatment of Minor and Major Bleeding Episodes
Table 8-123: Ixiaro - Recommended Dose, Cycle per Age & Cost per Cycle (US$)
Table 8-124: Cinryze - Recommended Dosing in Adults, Adolescents & Pediatric Patients
Table 8-125: Dysport - Dilution Instructions
Table 8-126: Dysport - Recommended Dosing by Muscle for Lower Limb Spasticity in Pediatric Patients
Table 8-127: Xenpozyme - Recommended Dose Escalation Regimen for Adult Patients
Table 8-128: Xenpozyme - Recommended Dose Escalation Regimen for Pediatric Patients
Table 8-129: Targretin - Initial Dose calculation According to Body Surface Area
Table 8-130: Dificid - Weight Dependent Recommended Dosage of Oral Suspension in Pediatric Patients
Table 8-131: Onfi - Recommended Total Daily Dose by Weight Group
Table 8-132: Noxafil - Dosing Regimens in Adult Patients
Table 8-133: Noxafil Delayed-Release Tablet & Noxafil Injection - Dosing Regimens for Pediatric Patients
Table 8-134: Noxafil Oral Suspension - Dosing Regimens for Pediatric Patients
Table 8-135: Envarsus RX - Recommended Tacrolimus Whole Blood Trough Concentration Ranges in Kidney Transplant Patients with Antibody Induction
Table 8-136: Albenza - Recommended Dosage
Table 8-137: Trisenox - Recommended Dosage in Combination With Tretinoin
Table 8-138: Ammonul - Recommended Dosage & Administration
Table 8-139: Cuvposa - Recommended Dose Titration Schedule (each dose to be given three times daily)
Table 8-140: BioThrax - Recommended Dose for Pre-Exposure Prophylaxis
Table 8-141: BioThrax - Recommended Dose for Post-Exposure Prophylaxis
Table 8-142: Hemangeol - Recommended Dose Titration According to Weight
Table 8-143: Acetadote - Three-Bag Recommended Dosage & Dilution for Patients 5 kg or greater
Table 8-144: Acetadote - Two -Bag Recommended Dosage & Dilution for Patients 41 kg or Greater
Table 8-145: ATryn - Recommended Dosing Formula
Table 8-146: ATryn - Recommended AT Activity Monitoring and Dose Adjustment
Table 8-147: Totect - Recommended Dose & Maximum Daily Dose
Table 8-148: Cafcit - Recommended Loading Dose & Maintenance Dose
Table 8-149: Corifact -Dose Adjustment Using the Berichrom Activity Assay
Table 8-150: Diastat - Recommended Dosage per Age (Years)
Table 8-151: Diastat - Recommended Dose (mg) per Weight kg) & Age (Years)
Table 8-152: Cholbam - Number of Capsules Needed to Achieve a Recommended Dosage of 10 mg/kg/day
Table 8-153: Cholbam - Number of Cholbam Capsules Needed to Achieve a Recommended Dosage of 15 mg/kg/day
Table 8-154: US - lenalidomide Orphan Drug Designations
Table 8-155: Coagadex - Recommended Dose for Prophylaxis of Bleeding Episodes
Table 8-156: Coagadex - Recommended Dose for on-Demand Treatment & Control of Bleeding Episodes
Table 8-157: Coagadex - Recommended Dose for Peri-Operative Management of Bleeding
Table 8-158: Anavip - Recommended Dosage & Administration
Table 8-159: Anascorp - Recommended Dosage & Administration
Table 8-160: Nithiodote - Recommended Age Specific Dosing
Table 8-161: Sarclisa - Dosing Schedule in Combination With Pomalidomide & Dexamethasone or in Combination With Carfilzomib & Dexamethasone
Table 8-162: Sarclisa - Dosing Schedule in Combination With Bortezomib, Lenalidomide & Dexamethasone
Table 8-163: Alyftrek - Recommended Dosage in Adult and Pediatric Patients Aged 6 Years & Older
Table 8-164: Recommended Dosage of Levacetylleucine Based on Body Weight (kg)
Table 8-165: Rivfloza - Dose Regimen in Adults & Pediatric Patients
Table 8-166: Grafapex - Dosage Regimen for Therapy-Based Allogeneic HSCT
Table 8-167: Sohonos - Recommended Weight-Based Dosage for Pediatric Patients Aged 8 to 13 Years for Females &10 to 13 Years for Males
Table 8-168: Talvey - Weekly Dosing Schedule
Table 8-169: Talvey - Biweekly Dosing Schedule
Table 8-170: Penpulimab - Order of Administration & Regimen in Combination with Either Cisplatin or Carboplatin & Gemcitabine
Table 8-171: Tevimbra - Recommended Dosages as a Single Agent or in Combination with Other Therapeutic Agent
Table 8-172: Duvyzat - Recommended Dosage in Patients 6 Years of Age & Older for the Treatment of DMD
Table 8-173: Fexinidazole - Recommended Dosage of Patients 6 Years of Age & Older & Weighing At Least 20 kg
Table 8-174: Zilbrysq - Total Daily Dosage by Body Weight Range
Table 8-175: Toripalimab - FDA Orphan Drug Designation Dates
Table 8-176: Elrexfio - Dosing Schedule
Table 8-177: Crenessity - Recommended Weight-Based Dosage for Pediatric Patients 4 Years of Age & Older
Table 8-178: Revuforj - Recommended Dosage for Patients 1 Year & Older
Table 8-179: Revuforj - Recommended Dosage Using Tablets for Patients Weighing Less Than 40 kg
Table 8-180: Imdelltra - Recommended Dosage and Schedule
Table 8-181: Tecvayli - Dosing Schedule
Table 8-182: Vanflyta - Dosage Regimen
Table 8-183: Imjudo - Recommended Dosage in Combination with Durvalumab
Table 8-184: Ojemda - Recommended Tablets Dosage Based on Body Surface Area
Table 8-185: Ojemda - Recommended Dosage Oral Suspension Based on Body Surface Area
Table 8-186: Piasky - Dosage Regimen Based on Body Weight

Companies Mentioned (Partial List)

A selection of companies mentioned in this report includes, but is not limited to:

AbbVie
Alnylam Pharmaceuticals
Amgen
Amicus Therapeutics
Array BioPharma
Astellas Pharma
AstraZeneca
Bayer HealthCare
BioMarin Pharmaceutical
Bioverativ
Boehringer Ingelheim
Bristol-Myers Squibb
Chiesi
Chugai Pharmaceutical
Collaborations Pharmaceuticals
CSL
Daiichi Sankyo Company
Eisai Co Ltd
Eli Lilly & Company
Emergent BioSolutions
Ferring Pharmaceuticals
Flavocure Biotech
Genentech
Genmab
Gilead Sciences
GSK
Hanmi Pharmaceutical
Incyte Corporation
Ionis Pharmaceuticals
Ipsen
Jazz Pharmaceuticals
Johnson & Johnson
Kamada
Krystal Biotech
Kyowa Kirin
Leadiant Biosciences
Ligand Pharmaceuticals
Lundbeck
MeiraGTx
Merck
Moderna Therapeutics
Novartis
Novo Nordisk
Omeros Corporation
Onyx Pharmaceuticals (Amgen)
Otsuka Pharmaceutical
Pfizer
PTC Therapeutics
Recordati
Regeneron Pharmaceuticals
Roche
Sanofi
Servier
Shionogi
Sumitomo Pharma
Sutro Biopharma
Swedish Orphan Biovitrum
Takeda
Teva Pharmaceutical Industries
UCB
UniQure
Vertex Pharmaceuticals
XOMA

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U.S. Orphan Drug Market, Drug Sales, Price, Dosage & Clinical Pipeline Insight 2031

Report Findings & Highlights:

FDA Designated Orphan Drug Designation & Why This Report?

FDA Designated Orphan Drug Clinical Trials Insights Covered in the Report

Leading Companies Active in R&D on FDA Designated Orphan Drugs

Report Indicating the Future Development of FDA Designated Orphan Drugs

Table of Contents

Companies Mentioned (Partial List)

Report Findings & Highlights:

FDA Designated Orphan Drug Designation & Why This Report?

FDA Designated Orphan Drug Clinical Trials Insights Covered in the Report

Leading Companies Active in R&D on FDA Designated Orphan Drugs

Report Indicating the Future Development of FDA Designated Orphan Drugs

Table of Contents

Companies Mentioned (Partial List)

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