US Orphan Drugs Market, Drugs Sales, Price, Dosage & Clinical Trials Insight 2028

Report
3600 Pages
July 2023
Region: United States
Kuick Research
ID: 5850398

Report Highlights:

US Orphan Drugs Market Opportunity: > USD 150 Billion By 2028
Orphan Drugs Dosage, Price and Treatment Cost: > 350 Orphan Drug
Annual and Quarterly Sales Insight (2019 - Q1’2023):> 100 Orphan Drugs
US Orphan Drugs Reimbursement Scenario: Medicare, Medicaid, Private Insurers
Active Clinical Trials Insight By Company, Indication and Phase: 1000 Orphan Drugs
Marketed Orphan Drugs Clinical Insight By Company and Indication: > 400 Orphan Drugs
Competitive Landscape: 75 Companies

Considering the increasing burden of patients suffering from rare and orphan diseases, the US FDA has taken several steps to increase the interest of the research and pharmaceutical companies to these forgotten diseases. One of the fundamental actions taken by the FDA is the assignment of the orphan drug designation, which gives developers benefits in the course of the research and development, clinical assessment, and marketing of the drug. The orphan drug designation has been granted to over 6500 drugs and compounds, and around 100 of these have received regulatory approvals. Therefore, though the number is less, these drugs have been essential for improving the treatment outcomes of patients suffering from rare orphan diseases. Taking into account the continued success of several drugs in the US pharmaceutical market, it is evident these therapeutic products are faring well in spite of the small patient base, which makes it an interesting domain to explore.

Earlier rare diseases were neglected due to low profitability feasibility, but the enactment of FDA’s Orphan Drug Act 1983, succeeding regulatory reforms, government funding support, and extensive research by pharmaceutical companies led to exponential growth in the US orphan drugs market. These favorable parameters resulted in more than 1000 orphan-designated drugs in clinical trials and more than 400 orphan-designated drugs being commercially available in the market. This number is further expected to surpass 500 orphan-designated drugs in the market by 2028 driven by the increasing focus of pharmaceutical companies on rare diseases. The US orphan drugs market was valued at around US$ 100 Billion in 2022 and is further expected to surpass US$ 180 Billion by 2028, making it one of the highest revenue-generating segments of the global orphan-designated drugs market.

Many drugs that were given the orphan designation for rare indications have already gained US FDA approval for a different, non-orphan indication. Observation studies, anecdotal evidence, or unexpected clinical results from clinical trials involving patients with unrelated indications are what lead researchers to the conclusion that the same drug may also be utilized for a rare indication. As a result, it frequently happens that pharmaceutical companies are unaware of the potential of their products as therapeutic treatments for rare diseases. On the plus side, completing clinical trials for uncommon diseases, and subsequently being given the orphan drug designation, can aid in growing the patient base for the company and the drug, which will later boost revenues.

For instance, Eylea, which was initially approved in 2011 for the treatment of wet age-related macular degeneration, was given its first orphan drug designation for the treatment of retinopathy of prematurity in February 2023, and was immediately approved for the same indication. Eight approvals for Eylea have been received since 2011 before this recent new approval, which supports the fact that many pharmaceutical companies are unable to foresee the potential of their products in orphan indications. This only emphasizes the importance of conducting clinical trials for orphan indications that have underlying characteristics similar to more prevalent diseases.

It has been observed that the majority of orphan drugs were developed by modest-scale biotechnology firms and then acquired by prominent pharmaceutical companies. When it comes to exploring understudied diseases and disorders, smaller biotechnology companies have some distinct benefits. Their founders often have better knowledge and experience conducting research and developing medicines for rare diseases. Additionally, these companies do not have the complex bureaucracy found in larger companies, which simplifies decision-making. In addition, they stand to lose less money if the project does not work out. Therefore, if their therapeutics are successful, the majority of these small biotechnology companies are bought by larger ones, greatly enhancing their credibility in the orphan drug market. On the same vein, larger companies are also known to purchase orphan drug candidates in exchange for royalties and milestone payments.

For instance, Amgen and Horizon Therapeutics reached a deal in December 2022 for Amgen to buy the company and add Horizon’s orphan drugs Actimmune, Ravicti, Tepezza, and Uplizna to its pipeline. Although the deal has not yet been finalized, it presents Amgen with the chance to expand its assembly of orphan drugs for rare diseases.

In the past couple years, number of clinical candidates receiving the orphan drug designation have increased drastically, which can be attributed to the benefits offered by the FDA. Temferon, RAG-17, INB-400, Batiraxcept and ZYIL1 are only a few candidates that received the orphan drug designation in 2023 for a variety of rare diseases. It is expected that as more companies understand the unmet needs of patients suffering from rare diseases, more drug candidates for these indications will enter the pipeline. The US currently leads the sale and development of orphan drugs due to the existence of numerous companies dedicated to treating rare diseases, favorable government regulations, such as Medicare/Medicaid reimbursements, and the extensive network of patient advocacy groups that work to support those with rare diseases.

1. US Orphan Drugs Market Overview

1.1 FDA Designation Criteria & Market Exclusivity
1.2 Current Market Scenario
1.3 Future Market Trends & Opportunity Assessment

2. Partnerships & Collaborations Concerning Orphan Drugs

3. US Orphan Drugs Market Trends By Indication

3.1 Cancer
3.2 Neurological Disorders
3.3 Cardiovascular Disorders
3.4 Ophthalmic Diseases
3.5 Rare Genetic Disorders & Metabolic Disorders
3.6 Autoimmune & Inflammatory Diseases

4. US Orphan Drugs Reimbursement Scenario

4.1 Medicare
4.2 Medicaid
4.3 Private Insurers
4.4 Pharmaceutical Companies

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

A full list of the drugs mentioned is available in report sample.

5.1 Lynparza
5.2 Nplate
5.3 Tafinlar
5.4 Halaven
5.5 Jadenu
5.6 Xpovio
5.7 Gavreto
5.8 Ocaliva
5.9 INOmax
5.10 Truseltiq
5.11 Lumakras
5.12 Monjuvi
5.13 Onpattro
5.14 Opdivo
5.15 Opdualag
.
.
.
.

5.370 Colazal (Balsalazide Disodium)
5.371 Anascorp (Centruroides (Scorpion) Immune F(ab’)2 (Equine) Injection)
5.372 Nithiodote (sodium nitrite and sodium thiosulfate)
5.373 BabyBIG (Botulism Immune Globulin Intravenous [Human])
5.374 NexoBrid (anacaulase-bcdb)
5.375 Sarclisa (Isatuximab)

6. US Orphan Designated Drugs Sales Insight (2019 - Q1’2023)

A full list of the drugs mentioned is available in report sample.

6.1 Tafinlar
6.2 Jadenu
6.3 Zolgensma
6.4 Lynparza
6.5 Nplate
6.6 Lumakras
6.7 Repatha
6.8 Otezla
6.9 Kyprolis
6.10 Opdivo
6.11 Opdualag
6.12 Empliciti
6.13 Braftovi
6.14 Mektovi
6.15 Lorbrena
.
.
.
.
6.100 Praluent
6.101 Mozobil
6.102 Yescarta
6.103 Lunsumio
6.104 Keytruda
6.105 Pemazyre
6.106 Camzyos

7. US Orphan Designated Drugs Clinical Trials Insight

7.1 By Company
7.2 By Indication
7.3 By Patient Segment
7.4 By Phase
7.5 By Priority Status

8. US Orphan Drugs Clinical Trials By Company, Indication & Phase

8.1 Research
8.2 Preclinical
8.3 Phase-I
8.4 Phase-I/II
8.5 Phase-II
8.6 Phase-II/III
8.7 Phase-III
8.8 Preregistration
8.9 Registered

9. Marketed Orphan Drugs Clinical Insight By Company & Indication

10. Competitive Landscape

10.1 AbbVie
10.2 Alexion (AstraZeneca Rare Disease Group)
10.3 Alnylam Pharmaceuticals
10.4 Amgen
10.5 Amicus Therapeutics
10.6 Array BioPharma
10.7 Astellas Pharma
10.8 AstraZeneca
10.9 Bayer HealthCare
10.10 Beacon Therapeutics
10.11 BioMarin Pharmaceutical
10.12 Bioverativ
10.13 Boehringer Ingelheim
10.14 Bristol-Myers Squibb
10.15 Celgene Corporation
10.16 Chiesi
10.17 Chugai Pharmaceutical
10.18 Collaborations Pharmaceuticals
10.19 CSL
10.20 Daiichi Sankyo Company
10.21 Eisai Co Ltd
10.22 Eli Lilly & Company
10.23 Emergent BioSolutions
10.24 Ferring Pharmaceuticals
10.25 Fred Hutchinson Cancer Research Center
10.26 Flavocure Biotech
10.27 Genentech
10.28 Genmab
10.29 Gilead Sciences
10.30 GlaxoSmithKline
10.31 GSK
10.32 Hanmi Pharmaceutical
10.33 Horizon Therapeutics plc
10.34 Incyte Corporation
10.35 Ionis Pharmaceuticals
10.36 Ipsen
10.37 Janssen Biotech
10.38 Jazz Pharmaceuticals plc
10.39 Johnson & Johnson
10.40 Kamada
10.41 Krystal Biotech
10.42 Kyowa Kirin
10.43 Leadiant Biosciences
10.44 Ligand Pharmaceuticals
10.45 Lundbeck A/S
10.46 MeiraGTx
10.47 Memorial Sloan-Kettering Cancer Center
10.48 Merck
10.49 Moderna Therapeutics
10.50 Nationwide Children's Hospital
10.51 Nonindustrial Source
10.52 Novartis
10.53 Novo Nordisk
10.54 Omeros Corporation
10.55 Onyx Pharmaceuticals (Amgen)
10.56 Otsuka Pharmaceutical
10.57 Pfizer
10.58 PTC Therapeutics
10.59 Recordati
10.60 Regeneron Pharmaceuticals
10.61 Roche
10.62 Sanofi
10.63 Servier
10.64 Shionogi
10.65 Spark Therapeutics
10.66 Sumitomo Pharma
10.67 Sutro Biopharma
10.68 Swedish Orphan Biovitrum
10.69 Takeda
10.70 Teva Pharmaceutical Industries
10.71 UCB
10.72 UniQure
10.73 Vertex Pharmaceuticals
10.74 XOMA
10.75 ZymoGenetics (BMS)

List of Figures

Figure 1-1: US - Orphan Drugs Market Size (US$ Billion), 2022 - 2028
Figure 3-1: TP-1287 - Study Initiation and Completion Year
Figure 3-2: ATG-022 - Study Initiation and Completion Year
Figure 3-3: ATG-101: Study Initiation and Completion Year
Figure 3-4: Lacutamab - Study Initiation and Completion Year
Figure 3-5: CART-ddBCMA - Study Initiation and Completion Year
Figure 3-6: FORE8394 - Study Initiation and Completion Year
Figure 3-7: ERAS-801 - Study Initiation and Completion Year
Figure 3-8: Tirabrutinib - Study Initiation and Completion Year
Figure 3-9: Temferon - Study Initiation and Completion Year
Figure 3-10: RP-A601 - Study Initiation and Completion Year
Figure 3-11: RP-A501 - Study Initiation and Completion Year
Figure 3-12: HG004 - Study Initiation and Completion Year
Figure 3-13: EA-2353 - Study Initiation and Completion Year
Figure 3-14: ARD-101 - Study Initiation and Completion Year
Figure 3-15: SYNB1934 - Study Initiation and Completion Year
Figure 3-16: ST-920 - Study Initiation and Completion Year
Figure 5-1: US - Lynparza Approvals by Indication
Figure 5-2: US - Lynparza Orphan Drug Designation Years by Indication
Figure 5-3: US - Cost of 60 and 120 Tablets Supply of 100 mg and 150 mg Lynparza(US$), July’2023
Figure 5-4: US - Monthly Cost of Treatment with 100 mg and 150 mg Lynparza Tablets (US$), July’2023
Figure 5-5: US - Cost of 125 mcg, 250 mcg and 500 mcg Supply Packs of Nplate (US$), July’2023
Figure 5-6: US - Tafinlar Approvals by Indication
Figure 5-7: US - Tafinlar Orphan Drug Designation Years
Figure 5-8: US - Cost of Per Unit and Supply of 120 Capsules of 50mg and 75mg Tafinlar (US$), July’2023
Figure 5-9: US - Monthly Cost of Tafinlar Treatment by Weight (US$), July’2023
Figure 5-10: US - Halaven Approvals by Indication
Figure 5-11: US - Monthly Cost of Treatment with Havalen Injection (US$), July’2023
Figure 5-12: US - Jadenu Orphan Drug Designation Years by Indication
Figure 5-13: US - Cost of Per Unit and Supply of 30 Tablets of 90mg, 180mg and 360mg Jadenu (US$), July’2023
Figure 5-14: US - Monthly Cost of Treatment with Jadenu (US$), July’2023
Figure 5-15: US - Xpovio Orphan Drug Designation and Approval Years by Indication
Figure 5-16: US - Cost of Per Unit and Supply of 20mg Xpovio Tablets (US$), July’2023
Figure 5-17: US - Cost of Per Unit and Supply of 40mg Xpovio Tablets (US$), July’2023
Figure 5-18: US - Cost of Per Unit and Supply of 50mg and 60mg Xpovio Tablets (US$), July’2023
Figure 5-19: US - Monthly Cost of Treatment with Xpovio (US$), July’2023
Figure 5-20: US - Gavreto Orphan Drug Designation and Approval Years by Indication
Figure 5-21: US - Cost of Per Unit and Supply of 100mg Gavreto Tablets (US$),July’2023
Figure 5-22: US - Monthly Cost of Treatment with Gavreto Capsule (US$), July’2023
Figure 5-23: US - Cost of Per Unit and Supply of 5mg and 10mg Ocaliva Tablets (US$), July’2023
Figure 5-24: US - INOmax Orphan Drug Designation and Approval Years
Figure 5-25: US - Truseltiq Orphan Drug Designation and Approval Years
Figure 5-26: US - Cost of Per Unit and Supply of Different Truseltiq Doses (US$), July’2023
Figure 5-27: US - Monthly and Yearly Cost of Treatment with Truseltiq (US$), July’2023
Figure 5-28: US - Lumakras Orphan Drug Designation and Approval Years
Figure 5-29: US - Cost of Per Unit and Supply of 120mg and 320mg Lumakras Doses (US$), July’2023
Figure 5-30: US - Monjuvi Orphan Drug Designation and Approval Years by Indication
Figure 5-31: US - Cost of Truseltiq Treatment Cycles US$), July’2023
Figure 5-32: US - Onpattro Orphan Drug Designation and Approval Years
Figure 5-33: US - Cost of Per Unit and Supply of 5mL Vial of Onpattro (US$), July’2023
Figure 5-34: US - Cost of Onpattro Treatment by Weight US$), July’2023
Figure 5-35: US - Cost of Per Unit and Supply of 10 mg/mL Opdivo (US$), July’2023
Figure 5-36: US - Opdualag Orphan Drug Designation and Approval Years
Figure 5-37: US - Cost of Per Unit and Supply of Opdualag Solution (US$), July’2023
Figure 5-38: US - Unit, Monthly and Yearly Cost of Opdualag Treatment US$), July’2023
Figure 5-39: US - Orladeyo Orphan Drug Designation and Approval Years
Figure 5-40: US - Cost of Per Unit and Supply of Orladeyo Capsule (US$), July’2023
Figure 5-41: US - Orladeyo Orphan Drug Designation and Approval Years
Figure 5-42: US - Cost of Per Unit and Supply of Rubraca Tablet (US$), July’2023
Figure 5-43: US - Livmarli Orphan Drug Designation and Approval Years
Figure 5-44: US - Cost of Per Unit and Supply of Livmarli Solution (US$), July’2023
Figure 5-45: US - Unit, Monthly and Yearly Cost of Livmarli Treatment US$), July’2023
Figure 5-46: US - Bendeka Orphan Drug Designation and Approval Years
Figure 5-47: US - Cost of Per Unit and Supply of Bendeka Solution (US$), July’2023
Figure 5-48: US - Unit Cost and Cost of 1 and 6 Cycles of Bendeka Treatment for CLL US$), July’2023
Figure 5-49: US - Unit Cost and Cost of 1 and 6 Cycles of Bendeka Treatment for CLL US$), July’2023
Figure 5-50: US - Ryanodex Orphan Drug Designation and Approval Years
Figure 5-51: US - Monthly Cost of Treatment with Fyarro Injection (US$), July’2023
Figure 5-52: US - Firdapse Orphan Drug Designation and Approval Years
Figure 5-53: US - Cost of Per Unit and Supply of 10mg Firdapse Tablet (US$), July’2023
Figure 5-54: US - Braftovi Orphan Drug Designation and Approval Years
Figure 5-55: US - Cost of Per Unit and Supply of 75mg Braftovi Capsule (US$), July’2023
Figure 5-56: US - Monthly Cost of Melanoma Treatment with Braftovi Capsule (US$), July’2023
Figure 5-57: US - Monthly Cost of Colorectal Cancer Treatment with Braftovi Capsule (US$), July’2023
Figure 5-58: US - Cost of Per Unit and Supply of 15mg Mektovi Tablet (US$), July’2023
Figure 5-59: US - Monthly Cost of Melanoma Treatment with Mektovi Tablet (US$), July’2023
Figure 5-60: US - Evrysdi Orphan Drug Designation and Approval Years
Figure 5-61: US - Cost of Per Unit and Supply of 0.75mg/mL Evrysdi Solution (US$), July’2023
Figure 5-62: US - Hetlioz Orphan Drug Designation and Approval Years by Indication
Figure 5-63: US - Cost of Per Unit and Supply of 20mg Hetlioz Capsule (US$), July’2023
Figure 5-64: US - Cost of Per Unit and Supply of 20mg Hetlioz LQ Oral Suspension(US$), July’2023
Figure 5-65: US - Monthly Cost of Melanoma Treatment with Hetlioz Capsule (US$), July’2023
Figure 5-66: Yervoy - First Approval by Indication
Figure 5-67: US - Cost of Per Unit and Supply of Yervoy Solution (US$), July’2023
Figure 5-68: US - Lorbrena Orphan Drug Designation and Approval Years
Figure 5-69: US - Cost of Per Unit and Supply of Lorbrena Tablet (US$), July’2023
Figure 5-70: US - Monthly and Yearly Cost of Treatment with Lorbrena Tablet (US$), July’2023
Figure 5-71: US - Terlivaz Orphan Drug Designation and Approval Years
Figure 5-72: US - Brukinsa Orphan Drug Designation and Approval Years by Indication
Figure 5-73: US - Cost of Per Unit and Supply of Brukinsa Capsule (US$), July’2023
Figure 5-74: US - Monthly and Yearly Cost of Treatment with Brukinsa (US$), July’2023
Figure 5-75: US - Viltepso Orphan Drug Designation and Approval Years
Figure 5-76: US - Monthly and Yearly Cost of Treatment with Viltepso (US$), July’2023
Figure 5-77: US - Eylea Orphan Drug Designation and Approval Years
Figure 5-78: US - Cost of Per Unit and Supply of Eylea Solution (US$), July’2023
Figure 5-79: US - Monthly and Yearly Cost of Treatment with Viltepso (US$), July’2023
Figure 5-80: US - Evkeeza Orphan Drug Designation and Approval Years
Figure 5-81: US - Cost of Per Unit and Supply of Evkeeza Solution (US$), July’2023
Figure 5-82: US - Sogroya Orphan Drug Designation and Approval Years
Figure 5-83: US - Cost of Per Unit and Supply of Sogroya Solution (US$), July’2023
Figure 5-84: US - Skytrofa Orphan Drug Designation and Approval Years
Figure 5-85: US - Cost of Per Unit and Supply of Skytrofa Injection (US$), July’2023
Figure 5-86: US - Cost of Per Unit and Supply of Avastin Solution (US$), July’2023
Figure 5-87: US - Vyvgart Orphan Drug Designation and Approval Years
Figure 5-88: US - Cost of Per Unit and Supply of Vyvgart Solution (US$), July’2023
Figure 5-89: US - Monthly Cost of Treatment with Vyvgart (US$), July’2023
Figure 5-90: US - Tpoxx Orphan Drug Designation and Approval Years
Figure 5-91: US - Koselugo Orphan Drug Designation and Approval Years
Figure 5-92: US - Cost of Per Unit and Supply of 10mg Capsules of Koselugo (US$), July’2023
Figure 5-93: US - Cost of Per Unit and Supply of 25mg Capsules of Koselugo (US$), July’2023
Figure 5-94: US - Monthly Cost of Treatment with Koselugo by BSA (US$), July’2023
Figure 5-95: US - Actemra Orphan Drug Designation and Approval Years
Figure 5-96: US - Cost of Per Unit and Supply of 20 mg/mL Vials of Actemra IVSolution (US$), July’2023
Figure 5-97: US - Cost of Per Unit and Supply of 162 mg/0.9 mL Prefilled Syringe of Actemra Subcutaneous Solution (ACTpen) (US$), July’2023
Figure 5-98: US - Tecentriq Orphan Drug Designation and Approval Years
Figure 5-99: US - Cost of Per Unit and Supply of Tecentriq Intravenous Solution (US$),July’2023 Figure 5-100: US - Qinlock Orphan Drug Designation and Approval Years
Figure 5-101: US - Cost of Per Unit and Supply of Qinlock Tablets (US$), July’2023
Figure 5-102: US - Unit, Monthly and Annual Cost of Treatment with Qinlock (US$), July’2023
Figure 5-103: US - Wakix Orphan Drug Designation and Approval Years
Figure 5-104: US - Cost of Per Unit and Supply of Wakix Tablets (US$), July’2023
Figure 5-105: US - Kimmtrak Orphan Drug Designation and Approval Years
Figure 5-106: US - Cost of Per Unit and Supply of Kimmtrak (US$), July’2023
Figure 5-107: US - Elahere Orphan Drug Designation and Approval Years
Figure 5-108: US - Cost of Per Unit and Supply of 100 mg/20 mL Elahere Solution (US$), July’2023
Figure 5-109: US - Price for Single Unit of Polivy 30 mg and 140 mg Intravenous Powder for Injection (US$), July’2023
Figure 5-110: US - Cost of Single Dose, Single Treatment Cycle and Full Treatment of Hairy Cell Leukemia with 30 mg Polivy Powder (US$), July’2023
Figure 5-111: US - Cost of Single Dose, Single Treatment Cycle and Full Treatment of Hairy Cell Leukemia with 140 mg Polivy Powder (US$), May’2023
Figure 5-112: US - Enhertu Orphan Drug Designation and Approval Years
Figure 5-113: Enhertu - Cost of Single Treatment Cycle and Annual Treatment (US$), July’2023
Figure 5-114: US - Jakafi Orphan Drug Designation and Approval Years
Figure 5-115: US - Cost of Per Unit and Supply of Different Jakafi Doses (US$), July’2023
Figure 5-116: Jakafi - Cost of Monthly and Annual Treatment (US$), July’2023
Figure 5-117: US - Kalydeco Orphan Drug Designation and Approval Years
Figure 5-118: US - Cost of Per Unit and Supply of Kalydeco Oral Granule and Tablet (US$), July’2023
Figure 5-119: Kalydeco - Cost of Monthly and Annual Treatment (US$), July’2023
Figure 5-120: US - Cost of Per Unit and Supply of Symdeko Oral Tablet (US$), July’2023
Figure 5-121: US - Trikafta Orphan Drug Designation and Approval Years
Figure 5-122: US - Cost of Per Unit and Supply of Trikafta Oral Granule and Tablet (US$), July’2023
Figure 5-123: US - Orkambi Orphan Drug Designation and Approval Years
Figure 5-124: US - Cost of Per Unit and Supply of Orkambi Oral Granule and Tablet (US$), July’2023
Figure 5-125: US - Hemgenix Orphan Drug Designation and Approval Years
Figure 5-126: US - Voxzogo Orphan Drug Designation and Approval Years
Figure 5-127: US - Cost of Per Unit and Supply of Voxzogo Powder (US$), July’2023
Figure 5-128: US - Actimmune Orphan Drug Designation and Approval Years
Figure 5-129: US - Cost of Per Unit and Supply of Actimmune Solution (US$), July’2023
Figure 5-130: US - Actimmune Orphan Drug Designation and Approval Years
Figure 5-131: US - Cost of per Unit of Reblozyl powder (US$), July’2023
Figure 5-132: US - Monthly and Annual Cost of Treatment with Reblozyl (US$), July’2023
Figure 5-133: US - Repatha Orphan Drug Designation and Approval Years
Figure 5-134: US - Cost of Per Unit and Supply of Repatha (US$), July’2023
Figure 5-135: US - Monthly and Annual Cost of Repatha Treatment in Adults with Established Cardiovascular Disease or Primary Hyperlipidemia (US$), July’2023
Figure 5-136: US - Monthly and Annual Cost of Repatha Treatment for HoFH (US$), July’2023
Figure 5-137: US - Rebyota Orphan Drug Designation and Approval Years
Figure 5-138: US - Cost of Per Unit and Supply of Rebyota (US$), July’2023
Figure 5-139: US - Cayston Orphan Drug Designation and Approval Years
Figure 5-140: US - Cost of Per Unit and Supply of Cayston Powder (US$), July’2023
Figure 5-141: US - Sylvant Orphan Drug Designation and Approval Years
Figure 5-142: US - Cost of Per Unit and Supply of Sylvant Powder (US$), July’2023
Figure 5-143: US - Cost of Monthly and Yearly Treatment with Sylvant US$), July’2023
Figure 5-144: US - Mylotarg Orphan Drug Designation and Approval Years
Figure 5-145: US - Cost of Per Unit and Supply of Mylotarg Powder (US$), July’2023
Figure 5-146: US - Cost of Induction Cycle, Consolidation Cycles and Full Treatment Cost of Newly Diagnosed De Novo CD33-positive AML with Mylotarg (US$), July’2023
Figure 5-147: US - Cost of Induction Phase and Continuation Phase and Full Treatment Cost of CD-33 positive AML with Mylotarg (US$), July’2023
Figure 5-148: US - Cost of Single Dose and Full Treatment Cost of Relapsed and Refractory CD33 Positive AML with Mylotarg (US$), July’2023
Figure 5-149: US - Besremi Orphan Drug Designation and Approval Years
Figure 5-150: US - Cost of Single Dose, Monthly and Yearly Treatment with Besremi (US$), July’2023
Figure 5-151: US - Ravicti Orphan Drug Designation and Approval Years
Figure 5-152: US - Cost of Per Unit and Supply of Ravicti (US$), July’2023
Figure 5-153: US - Cost of Per Unit and Supply of Tazverik Tablet (US$), July’2023
Figure 5-154: US - Luxturna Orphan Drug Designation and Approval Years
Figure 5-155: US - Zynteglo Orphan Drug Designation and Approval Years
Figure 5-156: US - Empaveli Orphan Drug Designation and Approval Years
Figure 5-157: US - Cost of Per Unit and Supply of Empaveli Solution (US$), July’2023
Figure 5-158: US - Cost of Single Dose, Monthly and Yearly Treatment with Empaveli(US$), July’2023
Figure 5-159: US - Ninlaro Orphan Drug Designation and Approval Years
Figure 5-160: US - Cost of Per Unit and Supply of Ninlaro Capsule (US$), July’2023
Figure 5-161: US - Cost of Single Dose, Monthly and Yearly Treatment with Empaveli (US$), July’2023
Figure 5-162: US - Otezla Orphan Drug Designation and Approval Years
Figure 5-163: US - Cost of Per Unit and Supply of Otezla tablet (US$), July’2023
Figure 5-164: US - Arzerra Orphan Drug Designation and Approval Years
Figure 5-165: US - Cost of Per Unit and Supply of Arzerra Solution (US$), July’2023
Figure 5-166: US - Macrilen Orphan Drug Designation and Approval Years
Figure 5-167: US - Cost of Per Unit and Supply of Macrilen Granules (US$), July’2023
Figure 5-168: US - Tagrisso Orphan Drug Designation and Approval Years
Figure 5-169: US - Cost of Per Unit and Supply of Tagrisso Tablets (US$), July’2023
Figure 5-170: US - Tagrisso Monthly, Yearly and Full Course Treatment Cost (US$), July’2023
Figure 5-171: US - Gammagard Liquid Orphan Drug Designation and Approval Years
Figure 5-172: US - Cost of Per Unit and Supply of Different Gammagard Liquid Vials (US$), July’2023
Figure 5-173: US - Fintepla Orphan Drug Designation and Approval Years
Figure 5-174: US - Cost of Per Unit and Supply of Fintepla Solution (US$), July’2023
Figure 5-175: US - Daybue Orphan Drug Designation and Approval Years
Figure 5-176: US - Takhzyro Orphan Drug Designation and Approval Years
Figure 5-177: US - Cost of Per Unit and Supply of Takhzyro Solution (US$), July’2023
Figure 5-178: US - Cyramza Orphan Drug Designation and Approval Years
Figure 5-179: US - Cost of Per Unit and Supply of Cyramza Solution (US$), July’2023
Figure 5-180: US - Hyftor Orphan Drug Designation and Approval Years
Figure 5-181: US - Cost of Per Unit and Supply of Hyftor Gel (US$), July’2023
Figure 5-182: US - Prevymis Orphan Drug Designation and Approval Years
Figure 5-183: US - Cost of Per Unit and Supply of Prevymis Solution (US$), July’2023
Figure 5-184: US - Cost of Per Unit and Supply of Prevymis Tablets (US$), July’2023
Figure 5-185: US - Prevymis Orphan Drug Designation and Approval Years
Figure 5-186: US - Cost of Initial, Subsequent and Annual Treatment with Zynlonta(US$), July’2023
Figure 5-187: US - Copiktra Orphan Drug Designation and Approval Years
Figure 5-188: US - Cost of Per Unit and Supply of Copiktra Capsules (US$), July’2023
Figure 5-189: US - Cost of Monthly and Annual Treatment with Copiktra (US$), July’2023
Figure 5-190: US - Tepezza Orphan Drug Designation and Approval Years
Figure 5-191: US - Cost of Single, Initial and Subsequent Doses of Copiktra (US$), July’2023
Figure 5-192: US - Mycapssa Orphan Drug Designation and Approval Years
Figure 5-193: US - Cost of Per Unit and Supply of Mycapssa Capsules (US$), July’2023
Figure 5-194: US - Juxtapid Orphan Drug Designation and Approval Years
Figure 5-195: US - Cost of Per Unit and Supply of Juxtapid Capsules (US$), July’2023
Figure 5-196: US - Myalept Orphan Drug Designation and Approval Years
Figure 5-197: US - Jelmyto Orphan Drug Designation and Approval Years
Figure 5-198: US - Mitosol Orphan Drug Designation and Approval Years
Figure 5-199: US - Cost of Per Unit and Supply of Mitosol Kit (US$), July’2023
Figure 5-200: US - Empliciti Orphan Drug Designation and Approval Years
Figure 5-201: US - Cost of Per Unit and Supply of Empliciti Powders (US$), July’2023
Figure 5-202: Empliciti - Cost of Treatment (US$), July’2023
Figure 5-203: US - Kcentra Orphan Drug Designation and Approval Years
Figure 5-204: US - Kyprolis Orphan Drug Designation and Approval Years by Indications
Figure 5-205: US - Cost Per Supply of 10mg, 30mg and 60mg Kyprolis (US$), July’2023
Figure 5-206: US - Enspryng Orphan Drug Designation and Approval Years by Indications
Figure 5-207: US - Cost of Treatment with Enspryng (US$), July’2023
Figure 5-208: US - Sirturo Orphan Drug Designation and Approval Years by Indications
Figure 5-209: US - Cost of Per Unit and Supply of 20mg and 100mg Sirturo Tablets (US$), July’2023
Figure 5-210: US - Tavalisse Orphan Drug Designation and Approval Years by Indications
Figure 5-211: US - Cost of Per Unit and Supply of Tavalisse Tablets (US$), July’2023
Figure 5-212: US - Gleolan Orphan Drug Designation and Approval Years by Indications
Figure 5-213: US - Keveyis Orphan Drug Designation and Approval Years
Figure 5-214: US - Cost of Per Unit and Supply of Keveyis Tablets (US$), July’2023
Figure 5-215: US - Zolgensma Orphan Drug Designation and Approval Years
Figure 5-216: US - Darzalex Orphan Drug Designation and Approval Years
Figure 5-217: US - Cost of Per Unit and Supply of Darzalex Solution (US$), July’2023
Figure 5-218: US - Darzalex Orphan Drug Designation and Approval Years
Figure 5-219: US - Doptelet Orphan Drug Designation and Approval Years
Figure 5-220: US - Cost of Per Unit and Supply of Doptelet Tablet (US$), July’2023
Figure 5-221: US - Endari Orphan Drug Designation and Approval Years
Figure 5-222: US - Cost of Per Unit and Supply of Endari Powder (US$), July’2023
Figure 5-223: US - NutreStore Orphan Drug Designation and Approval Years
Figure 5-224: US - Cost of Per Unit and Supply of NutreStore (US$), July’2023
Figure 5-225: US - Lymphoseek Orphan Drug Designation and Approval Years
Figure 5-226: US - Krystexxa Orphan Drug Designation and Approval Years
Figure 5-227: US - Cost of Treatment with Krystexxa (US$), July’2023
Figure 5-228: US - Krystexxa Orphan Drug Designation and Approval Years
Figure 5-229: US - Cost of Per Unit and Supply of Korlym (US$), July’2023
Figure 5-230: US - Monthly and Yearly Korlym Treatment Cost (US$), July’2023
Figure 5-231: US - Kalbitor Orphan Drug Designation and Approval Years
Figure 5-232: US - Livtencity Orphan Drug Designation and Approval Years
Figure 5-233: US - Cost of Per Unit and Supply of Livtencity (US$), July’2023
Figure 5-234: US - Monthly and Yearly Korlym Treatment Cost (US$), July’2023
Figure 5-235: US - Enjaymo Orphan Drug Designation and Approval Years
Figure 5-236: US - Cost of Per Unit and Supply of Enjaymo (US$), July’2023
Figure 5-237: US - Rethymic Orphan Drug Designation and Approval Years
Figure 5-238: US - Coartem Orphan Drug Designation and Approval Years
Figure 5-239: US - Cost of Per Unit and Supply of Coartem (US$), July’2023
Figure 5-240: US - Nulibry Orphan Drug Designation and Approval Years
Figure 5-241: US - Inmazeb Orphan Drug Designation and Approval Years
Figure 5-242: US - Valtoco Orphan Drug Designation and Approval Years
Figure 5-243: US - Cost of Per Unit and Supply of Valtoco (US$), July’2023
Figure 5-244: US - Detectnet Orphan Drug Designation and Approval Years
Figure 5-245: US - Folotyn Orphan Drug Designation and Approval Years
Figure 5-246: US - Cost of Per Unit and Supply of Folotyn (US$), July’2023
Figure 5-247: US - Cost of Single Infusion and 6-Cycle Treatment with Folotyn US$), July’2023
Figure 5-248: US - Cystadane Orphan Drug Designation and Approval Years
Figure 5-249: US - Cost of Per Unit and Supply of Cystadane (US$), July’2023
Figure 5-250: US - Cystaran Orphan Drug Designation and Approval Years
Figure 5-251: US - Cost of Per Unit and Supply of Cystaran (US$), July’2023
Figure 5-252: US - Voraxaze Orphan Drug Designation and Approval Years
Figure 5-253: US - Cost of Treatment with Voraxaze (US$), July’2023
Figure 5-254: US - Mepron Orphan Drug Designation and Approval Years
Figure 5-255: US - Cost of Per Unit and Supply of Mepron (US$), July’2023
Figure 5-256: US - Vistogard Orphan Drug Designation and Approval Years
Figure 5-257: US - Cost of Per Unit and Supply of Mepron (US$), July’2023
Figure 5-258: US - Xuriden Orphan Drug Designation and Approval Years
Figure 5-259: US - Cost of Per Unit and Supply of Mepron (US$), July’2023
Figure 5-260: US - Xatmep Orphan Drug Designation and Approval Years
Figure 5-261: US - Cost of Per Unit and Supply of Xatmep (US$), July’2023
Figure 5-262: US - Purixan Orphan Drug Designation and Approval Years
Figure 5-263: US - Cost of Per Unit and Supply of Purixan (US$), July’2023
Figure 5-264: US - Cost of Per Unit and Supply of Erwinaze (US$), July’2023
Figure 5-265: US - Ascor Orphan Drug Designation and Approval Years
Figure 5-266: US - Cost of Per Unit and Supply of Ascor (US$), July’2023
Figure 5-267: US - Tepadina Orphan Drug Designation and Approval Years
Figure 5-268: US - Cost of Per Supply of Tepadina (US$), July’2023
Figure 5-269: US - BAT Orphan Drug Designation and Approval Years
Figure 5-270: US - Busulfex Orphan Drug Designation and Approval Years
Figure 5-271: US - Cost of Per Unit and Supply of Busulfex (US$), July’2023
Figure 5-272: US - Lipiodol Orphan Drug Designation and Approval Years
Figure 5-273: US - Cost of Per Unit and Supply of Lipiodol (US$), July’2023
Figure 5-274: US - Doxil Orphan Drug Designation and Approval Years
Figure 5-275: US - Cost of Per Unit and Supply of Doxil (US$), July’2023
Figure 5-276: US - Nymalize Orphan Drug Designation and Approval Years
Figure 5-277: US - Cost of Per Unit and Supply of 30 mg/5 mL Nymalize (US$), July’2023
Figure 5-278: US - Cost of Per Unit and Supply of 60 mg/10 mL Nymalize (US$), July’2023
Figure 5-279: US - Priftin Orphan Drug Designation and Approval Years
Figure 5-280: US - Cost of Per Unit and Supply of Priftin (US$), July’2023
Figure 5-281: US - Gralise Orphan Drug Designation and Approval Years
Figure 5-282: US - Cost of Per Unit and Supply of Priftin (US$), July’2023
Figure 5-283: US - Gralise Orphan Drug Designation and Approval Years
Figure 5-284: US - Cost of Per Unit and Supply of Galzin (US$), July’2023
Figure 5-285: US - NeoProfen Orphan Drug Designation and Approval Years
Figure 5-286: US - Cost of Per Unit and Supply of NeoProfen (US$), July’2023
Figure 5-287: US - Photofrin Orphan Drug Designation and Approval Years
Figure 5-288: US - Retisert Orphan Drug Designation and Approval Years

Companies Mentioned (Partial List)

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

AbbVie
Alexion (AstraZeneca Rare Disease Group)
Alnylam Pharmaceuticals
Amgen
Amicus Therapeutics
Array BioPharma
Astellas Pharma
AstraZeneca
Bayer HealthCare
Beacon Therapeutics
BioMarin Pharmaceutical
Bioverativ
Boehringer Ingelheim
Bristol-Myers Squibb
Celgene Corporation
Chiesi
Chugai Pharmaceutical
Collaborations Pharmaceuticals
CSL
Daiichi Sankyo Company
Eisai Co Ltd
Eli Lilly & Company
Emergent BioSolutions
Ferring Pharmaceuticals
Fred Hutchinson Cancer Research Center
Flavocure Biotech
Genentech
Genmab
Gilead Sciences
GlaxoSmithKline
GSK
Hanmi Pharmaceutical
Horizon Therapeutics plc
Incyte Corporation
Ionis Pharmaceuticals
Ipsen
Janssen Biotech
Jazz Pharmaceuticals plc
Johnson & Johnson
Kamada
Krystal Biotech
Kyowa Kirin
Leadiant Biosciences
Ligand Pharmaceuticals
Lundbeck A/S
MeiraGTx
Memorial Sloan-Kettering Cancer Center
Merck
Moderna Therapeutics
Nationwide Children's Hospital
Nonindustrial Source
Novartis
Novo Nordisk
Omeros Corporation
Onyx Pharmaceuticals (Amgen)
Otsuka Pharmaceutical
Pfizer
PTC Therapeutics
Recordati
Regeneron Pharmaceuticals
Roche
Sanofi
Servier
Shionogi
Spark Therapeutics
Sumitomo Pharma
Sutro Biopharma
Swedish Orphan Biovitrum
Takeda
Teva Pharmaceutical Industries
UCB
UniQure
Vertex Pharmaceuticals
XOMA
ZymoGenetics (BMS)

License	Format	Properties	Price
SINGLE USER LICENSE PDF	The product is a PDF.	This is a single user license, allowing one user access to the product.	€8834EUR$9,000USD£7,385GBP
ENTERPRISE LICENSE PDF	The product is a PDF.	This is an enterprise license, allowing all employees within your organization access to the product.	€14723EUR$15,000USD£12,308GBP

Report Highlights:

Table of Contents

Companies Mentioned (Partial List)

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About the Orphan Drug Market