Austin, June 20, 2025 (GLOBE NEWSWIRE) -- Orphan Drugs Market Size & Growth Analysis:
According to SNS Insider, the Orphan Drugs Market was valued at USD 190.87 billion in 2024 and is anticipated to reach USD 475.61 billion by 2032, growing at a CAGR of 12.11% during the forecast period (2025–2032). The United States alone accounted for USD 56.25 billion in 2024 and is projected to hit USD 123.48 billion by 2032. The primary growth catalysts include rising public and private investment in R&D, gene-based therapies, favorable reimbursement policies, and expanding patient registries.
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Market Overview
Orphan drugs for treating diseases that afflict less than 200,000 individuals in the U.S. are attracting more than ever before from regulators and pharmaceutical innovators. The FDA's Orphan Drug Act and complementary legislation from the EMA have spurred market action by providing advantages such as market exclusivity, fee reductions, tax credits, and expedited approval channels. These instruments greatly alleviate commercial risks involved in developing drugs for small patient bases.
Pharma giants and biotech companies are pouring into the space, drawn by large margins and longer terms of exclusivity. Concurrently, technologies like gene therapy, CRISPR, monoclonal antibodies, and cell-based treatments are allowing precision medicines with revolutionary results.
The U.S. is still at the forefront of orphan drug development, fueled by robust R&D spending, swift FDA approval times, and growing collaborations between academia, biopharma, and patient organizations. Government initiatives and expanded access avenues are making treatments more broadly available to those suffering from rare disease.
Major Players Analysis Listed in this Report are:
- Amgen Inc.
- Bayer AG
- F. Hoffmann-La Roche Ltd
- Alexion Pharmaceuticals Inc.
- Novo Nordisk A/S
- Novartis AG
- Bristol-Myers Squibb Company
- AstraZeneca
- Daiichi Sankyo Company Limited
- GlaxoSmithKline
Orphan Drugs Market Report Scope
| Report Attributes | Details |
| Market Size in 2024 | USD 190.87 billion |
| Market Size by 2032 | USD 475.61 billion |
| CAGR (2025–2032) | 12.11% |
| U.S. Market 2024 | USD 56.25 billion |
| U.S. Forecast by 2032 | USD 123.48 billion |
| Base Year | 2024 |
| Forecast Period | 2025–2032 |
| Key Regional Coverage | North America (US, Canada, Mexico), Europe (Germany, France, UK, Italy, Spain, Poland, Turkey, Rest of Europe), Asia Pacific (China, India, Japan, South Korea, Singapore, Australia, Rest of Asia Pacific), Middle East & Africa (UAE, Saudi Arabia, Qatar, South Africa, Rest of Middle East & Africa), Latin America (Brazil, Argentina, Rest of Latin America) |
Segment Analysis
By Therapy Type
The orphan drugs market was dominated by the oncology segment with a share of 38.7 % in 2024. This hegemony is due to the high level of unmet need and increasing prevalence of orphan cancers like glioblastoma and neuroendocrine tumors. Because of the fast-track approval paths - especially for cancer drugs - it’s tempting for drugmakers to target these types of treatments.
By Drug Type
Biologics led the market in 2024 with 72%, on account of better target specificity and high efficiency for the treatment of rare diseases. Monoclonal antibodies, gene therapies, and recombinant proteins have made a breakthrough in the development of orphan drugs by delivering disease-modifying results in contrast to the palliative effects.
By Distribution Channel
Hospital pharmacy services held the major share of the distribution side with a share of 58% globally in 2024. It is mainly because orphan drugs are generally liable to special management, administration by hospital workers, and patient follow-up, which are easier to perform in a hospital.
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Orphan Drugs Market Segmentation
By Therapy Type
- Oncology
- Hematology
- Neurology
- Endocrinology
- Cardiovascular
- Respiratory
- Immunotherapy
- Infectious Diseases
- Others
By Drug Type
- Biologics
- Non-biologics
By Distribution Channel
- Hospital Pharmacy
- Retail Pharmacy
- Online Pharmacy
- Others
Regional Analysis
North America led the orphan drugs market in 2024, driven by favorable government legislation like the Orphan Drug Act, faster approval processes, high healthcare expenditure, and the dominant presence of key biopharma companies. The U.S. holds the largest share due to its strong regulatory support, early diagnosis capabilities, and strategic collaborations in rare disease drug development.
Asia Pacific is the fastest-growing region, propelled by rising awareness, increasing diagnosis rates, and growing government focus on rare diseases in countries like Japan, China, and South Korea. The expanding biopharma infrastructure and investment in local orphan drug R&D also contribute to rapid regional growth.
Recent Developments (Orphan Drugs Market)
- March 2025 – FDA granted orphan drug designation to radiprodil (GRIN Therapeutics) for GRIN-related neurodevelopmental disorder; Phase 3 trial planned for 2025.
- August 2024 – NXP800 by Nuvectis Pharma received orphan drug designation for ARID1a-deficient ovarian and peritoneal cancers.
- April 2024 – Pfizer launched Elrexfio for relapsed/refractory multiple myeloma under orphan status in the U.S.
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Table of Contents – Major Key Points
1. Introduction
2. Executive Summary
3. Research Methodology
4. Market Dynamics Impact Analysis
5. Statistical Insights and Trends Reporting
6. Competitive Landscape
7. Orphan Drugs Market by Therapy Type
8. Orphan Drugs Market by Drug Type
9. Orphan Drugs Market by Distribution Channel
10. Regional Analysis
11. Company Profiles
12. Use Cases and Best Practices
13. Conclusion
About Us:
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