This industry analysis report tackles the major orphan drugs within the rare disease therapeutics market and encompasses the most prevalent and dominant therapies in this space. The market value for the current major orphan therapeutic space is analysed and forecast to 2025 with corresponding CAGR percentages. Market details are provided by drug type, therapeutic area, geography and also specific sales and forecast of the major drugs in the field. Key industry players and developments are covered along with the growing pipeline and clinical trials. Regional insights and approval trends are given, as are the strengths, opportunities and challenges of this growing market.
The scope of the report provides an in-depth analysis of the therapeutics space by value and forecast to 2025 that is subdivided into:
- Small molecules
- Antibodies
- Oligonucleotides
- Gene therapy
- Cell therapy
The report reviews the most dominant therapeutics in each sub-market and provides clinical evaluation, sales forecast and market share to 2025. It gives a comparison between the current market values of the main therapies and how the landscape will change by 2025 with the launch of new pipeline agents. This includes some of the main blockbusters such as
- Revlimid
- Trikafta
- Darzalex
- Spinraza
- Hemlibra
- Venclexta
- Soliris
- Adcetris
- Jakafi
- Lenvima
- Lynparza
- Ultomiris
- Yescarta
- Pomalyst
- Vyndaqel
- Ninlaro
- Liso-cel
Therefore the reader is provided with data on the current main dominant therapeutics in the area and also the drugs that will dominate the space by 2025. This analysis is provided for each sub-therapeutic area. Geographical breakdown analysis is further provided and is segmented into North America, Europe, Asia Pacific and the Rest of the World.
The scope of the report also includes the orphan drug market by indication and disease type with specific detail given to oncology, hematological disorders, CNS, respiratory, immunomodulation, cardiovascular, musculoskeletal, anti-infectives, endocrine and gastrointestinal disease.
Key players and company profiles in the rare disease market are provided including:
- Bristol Myers Squibb
- Roche/Genentech
- Alexion
- Janssen J&J
- Biogen
- Novartis
- AstraZeneca Merck
- Eisai
- Takeda
- Abbvie Genentech
- Gilead (Kite)
- Pfizer
- Vertex
- Argenx
The report also includes a detailed description of the following trends and market shapers:
- USA (FDA) and European (EMA) Orphan Drug Approvals
- Venture Funding Increasing Over Time
- Therapeutic Areas Dominating Approval
- Orphan Drug Designation Trends
- Repurposing Existing Therapeutics
- Japan Approval Trends
- South Korea Approval Trends
- China Approval Trends
The growing rare disease pipeline and clinical trial environment is evaluated with specific consideration given to:
- Small Molecules and Multiple Myeloma
- Gene and Cell Therapy in Melanoma
- Gene and Cell Therapy in ADA-SCID
- Gene and Cell Therapy agents in Hemophilia A and B
- CAR-T Therapy agents in Pancreatic Cancer
- CAR-T Therapy agents in Multiple Myeloma
- Gene and Cell Therapies in Duchenne Muscular Dystrophy
- RPE65 Genetic Retinal Mutation
- Rare Eye Disorders
- Spinal Muscular Atrophy
- Sickle Cell Anaemia
- Cystic Fibrosis
- Erythropoietic Protoporphyria
- Guillain-Barre Syndrome
- Scleroderma
- Alpha-1 Antitrypsin Deficiency
- Juvenile Idiopathic Arthritis
Table of Contents
1. Executive Summary
2. USA (FDA) and European (EMA) Orphan Drug Approvals
2.1 Venture Funding Increasing Over Time
2.2 Therapeutic Areas Dominating Approval
2.3 Orphan Drug Designation Trends
2.4 Repurposing Existing Therapeutics
2.5 Japan Approval Trends
2.6 South Korea Approval Trends
2.7 China Approval Trends
3. Rare Disease Market Analysis
3.1 Major Orphan Drug Market to 2025
3.2 Orphan Small Molecule Market Analysis to 2025
3.3 Orphan Antibody Market Analysis to 2025
3.4 Orphan Gene Therapy Market Analysis to 2025
3.5 Orphan Cell Therapy Market Analysis to 2025
3.6 Orphan Oligonucleotide Therapy Market Analysis to 2025
3.7 Geographical Breakdown of Rare Disease Market to 2025
3.8 Orphan Drug Market by Indication and Disease Type
3.9 Key Market Players
3.9.1 Bristol-Myers Squibb
3.9.2 Roche/Genentech
3.9.3 Alexion
3.9.4 J&J/Janssen Biotech
3.9.5 Biogen
3.9.6 Novartis
3.9.7 AstraZeneca
3.9.8 Eisai
3.9.9 Takeda
3.9.10 Abbvie
3.9.11 Gilead
3.9.12 Pfizer
3.9.13 Vertex Pharmaceuticals
3.9.14 Argenix
4. Pipeline Agents in Clinical Trials by Condition, Intervention and Phase
4.1 Select Clinical Trials Investigating Small Molecules and Multiple Myeloma
4.2 Select Clinical Trials Investigating Gene and Cell Therapy in Melanoma
4.3 Select Clinical Trials Investigating Gene and Cell Therapy in ADA-SCID
4.4 Current Clinical Trials Investigating the RPE65 Genetic Retinal Mutation
4.5 Select Clinical Trials involving Rare Eye Disorders and Orphan Agents
4.6 Select Clinical Trials involving Spinal Muscular Atrophy and Orphan Agents
4.7 Select Clinical Trials involving Sickle Cell Anaemia and Orphan Agents
4.8 Select Clinical Trials involving Gene and Cell Therapy agents in Hemophilia A and B
4.9 Select Phase III Clinical Trials in Cystic Fibrosis
4.10 Select Clinical Trials involving Gene and Cell Therapy agents in Cystic Fibrosis
4.11 Select Clinical Trials involving CAR-T Therapy agents in Pancreatic Cancer
4.12 Select Clinical Trials involving CAR-T Therapy agents in Multiple Myeloma
4.13 Select Clinical Trials involving Gene and Cell Therapies in Duchenne Muscular Dystrophy
4.14 Select Clinical Trials involving Oligonucleotide Therapies in Rare Diseases
4.15 Select Clinical Trials Investigating Therapeutics in Erythropoietic Protoporphyria
4.16 Select Clinical Trials Investigating Therapeutics in Guillain-Barre Syndrome
4.17 Select Phase III Clinical Trials Investigating Therapeutics in Scleroderma
4.18 Select Phase III Clinical Trials Investigating Therapeutics in Alpha-1 Antitrypsin Deficiency
4.19 Select Phase III Clinical Trials Investigating Therapeutics in Juvenile Idiopathic Arthritis
5. Pipeline Therapeutics Analysis
6. Future Outlook
List of Exhibits
Exhibit 2.1: Number of FDA and EMA Orphan Drug Approvals from 2001-2018
Exhibit 2.2: FDA Priority, Accelerated, Breakthrough and Fast Track Regulatory Reviews for Rare Disease from 2008-2017
Exhibit 2.3: EMA PRIME, Accelerated Assessment, Exceptional Circumstance or Conditional Marketing Approval Reviews for Rare Disease from 2000-2018
Exhibit 2.4: Venture Funding into Rare Disorders, Excluding Oncology, 2013-2018
Exhibit 2.5: FDA Orphan Drug Approvals by Therapeutic Area
Exhibit 2.6: EMA Orphan Drug Approvals by Therapeutic Area
Exhibit 2.7: Number of FDA Approved Rare Disease Designations by Drug Type 2017-2020
Exhibit 2.8: Number of FDA Applications for Orphan Designation 2020
Exhibit 2.9: Number of Orphan FDA Designated Agents 2020
Exhibit 2.10: Percentage of FDA Applications for Orphan Designation 2020
Exhibit 2.11: FDA Approved Agents for Rare Disease Indications 2020
Exhibit 2.12: FDA Approved Agents for Rare Disease Indications 2019
Exhibit 2.13: FDA Approved Agents for Rare Disease Indications 2018
Exhibit 2.14: FDA Approved Agents for Rare Disease Indications 2017
Exhibit 2.15: EMA Approved Rare Disease Therapies to Date
Exhibit 3.1: Major Rare Disease Therapeutic Market by Drug Class 2018-2025
Exhibit 3.2 Major Rare Disease Therapeutic Market by Small Molecules, Antibodies, Gene Therapies, Cell Therapies and Oligonucleotides, 2018-2025
Exhibit 3.3: Percentage Market Share of Rare Disease Drugs by Subtype, 2018 Vs 2025
Exhibit 3.4: Top Rare Disease Drug Sales ($ Billion) 2018
Exhibit 3.5: Top Rare Disease Drug Sales Forecast ($ Billion) 2025
Exhibit 3.6: Current Small Molecules Dominating the Rare Disease Market and Forecast to 2025
Exhibit 3.7: Future Small Molecules within the Rare Disease Market and Forecast to 2025
Exhibit 3.8: Small Molecule Orphan Drug Market 2018-2025
Exhibit 3.9: Current & Future Antibodies Dominating the Rare Disease Market and Forecast to 2025
Exhibit 3.10: Orphan Antibody Market Analysis 2018-2025
Exhibit 3.11: Current & Future Gene Therapies Dominating the Rare Disease Market and Forecast to 2025
Exhibit 3.12: Orphan Gene Therapy Market Analysis 2018-2025
Exhibit 3.13: Current & Future CART Cell Therapies Dominating the Rare Disease Market and Forecast to 2025
Exhibit 3.14: Orphan Cell Therapy Market Analysis 2018-2025
Exhibit 3.15: Current & Future Oligonucleotide Therapies Dominating the Rare Disease Market and Forecast to 2025
Exhibit 3.16 Orphan Oligonucleotide Therapy Market Analysis 2018-2025
Exhibit 4.1: Select Clinical Trials Investigating Small Molecules and Multiple Myeloma
Exhibit 4.2: Select Phase III Clinical Trials Investigating Small Antibodies and Multiple Myeloma
Exhibit 4.3: Select Clinical Trials Investigating Gene and Cell Therapy in Melanoma
Exhibit 4.4: Select Clinical Trials Investigating Gene and Cell Therapy in ADA-SCID
Exhibit 4.5: Current Clinical Trials Investigating the RPE65 Genetic Retinal Mutation
Exhibit 4.6: Select Clinical Trials involving Rare Eye Disorders and Orphan Agents
Exhibit 4.7: Select Clinical Trials involving Spinal Muscular Atrophy and Orphan Agents
Exhibit 4.8: Select Clinical Trials involving Sickle Cell Anaemia and Orphan Agents
Exhibit 4.9: Select Clinical Trials involving Gene and Cell Therapy agents in Hemophilia A/B
Exhibit 4.10: Select Phase III Clinical Trials in Cystic Fibrosis
Exhibit 4.11: Select Clinical Trials involving Gene and Cell Therapy agents in Cystic Fibrosis
Exhibit 4.12: Select Clinical Trials involving CAR-T Therapy agents in Pancreatic Cancer
Exhibit 4.13: Select Clinical Trials involving CAR-T Therapy agents in Multiple Myeloma
Exhibit 4.14: Select Clinical Trials involving Gene and Cell Therapies in Duchenne Muscular Dystrophy
Exhibit 4.15: Select Clinical Trials involving Oligonucleotide Therapies in Rare Diseases
Exhibit 4.16: Select Clinical Trials Investigating Therapeutics in Erythropoietic Protoporphyria
Exhibit 4.17: Select Clinical Trials Investigating Therapeutics in Guillain-Barre Syndrome
Exhibit 4.18: Select Phase III Clinical Trials Investigating Therapeutics in Scleroderma
Exhibit 4.19: Select Phase III Clinical Trials Investigating Therapeutics in Alpha-1 Antitrypsin Deficiency
Exhibit 4.20: Select Phase III Clinical Trials Investigating Therapeutics in Juvenile Idiopathic Arthritis
Exhibit 5.1: Total Number of Orphan Drugs in Phase I, II, III and NDA/BLA Clinical Programs
Exhibit 5.2: Number of Orphan Drugs (Oncology/Ex-Oncology) in Phase I, II, III and NDA/BLA Clinical Programs
Exhibit 5.3: Number of Therapies in Development for Rare Diseases
Exhibit 5.4: Top Selling Orphan Drugs Forecast to 2025
Exhibit 5.5: Top Orphan Drugs by Type and Mode of Action by 2025
Samples
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Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Abbvie Genentech
- Alexion
- Argenx
- AstraZeneca Merck
- Biogen
- Bristol Myers Squibb
- Eisai
- Gilead (Kite)
- Janssen J&J
- Novartis
- Pfizer
- Roche/Genentech
- Takeda
- Vertex
Methodology
Based on Locations in Europe and Asia, the analyst team are all PhD-level experts and industry-experienced professionals. They pool resources, contacts, business acumen, and technical experience to provide cutting edge insights for all of the reports. The senior analysts have at least ten years’ experience in major strategic corporations such as BCC research and TriMarkPublications. The methodologies are clearly defined from the outset. Initially, a number of clear objectives are set, e.g., to identify the market size, segmentation, key players, SWOT analysis, influential technologies, and business and economic environments:
- By Company
- By Geography (US, UK, EU, Asia)
- By Segment
- By Sub-market
Key Strengths, Weaknesses and Threats InfluencingLeading Player Position within the Market
- Technologies Driving the Market
- Top Fastest Growing Market Segments and Emerging Opportunities
- Top Pharmaceutical Companies within the IPM by Market Share and Revenue
- Comprehensive Product Portfolios, R&D Activity, and Pipeline Therapeutics
- M&A Activity and Future Strategies of Top Personalized Medicine Pharmacos
Following this, the analyst determines key financial data & business strategy information to give clients the most accurate information required to identify areas of profitable growth and what technical advantages are required in a competitive landscape such as company financials, sales & revenue figures.
Market analysis is initiated using primary research tools such as speaking directly with end-users, identifying their needs, and any un-met needs in the market place. This exploratory research identifies any specific requirements in the market and is tailored specifically to niche markets such as gene therapy, regenerative medicine, personalized medicine, targeted therapeutics and companion diagnostics, drug delivery systems, cosmetic surgery and services, cancer biomarkers and immunotherapy. At KellyScientific, we have a wide range of contacts within these niche areas that provide us with cutting edge insights into a marketplace that is beyond the reach of many. We also travel to international conferences quarterly to source new data and trends from global experts.
Secondary research performed by Kelly Scientific is meticulously scrutinized and analysed prior to integration into a final report. We only use validated and confirmed sources of information from company-specific corporate websites, government websites and documents, annual reports, press releases, international peer-reviewed scientific and medical journals, and research reports. Together both primary and secondary research and also unique insights from the chief analysts and editor alike provide the client with a report that exceeds its competitors.
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