United States Orphan Drugs Market 2025 | Growth Drivers, Trends & Market Forecast, Competitive Landscape & Investment Opportunities

Market Size and Growth

The Global Orphan Drugs Market reached US$ 223.76billion in 2023 and is expected to reach US$ 486.51 billion by 2032, growing at a CAGR of 9.1% during the forecast period 2024-2032.

Key Development:

United States: Recent Industry Developments

✅ In September 2025, Dyne Therapeutics received Orphan Drug Designation in Japan for DYNE-251, a gene therapy targeting Duchenne Muscular Dystrophy.

✅ In August 2025, Eisai's anticancer agent Tazemetostat received Orphan Drug Designation in Japan, aiming to address unmet needs in oncology.

✅ In July 2025, Arcus Biosciences' Quemliclustat was granted Orphan Drug Designation by the FDA for pancreatic cancer, qualifying for incentives like tax credits and market exclusivity.

Japan: Recent Industry Developments

✅ In September 2025, JR-446, an investigational drug for Mucopolysaccharidosis Type IIIB, was granted Orphan Drug Designation by Japan's Ministry of Health, Labour and Welfare.

✅ In August 2025, Japan committed new funding to develop treatments for drug-resistant infections, highlighting the nation's dedication to addressing rare and complex diseases.

✅ In July 2025, the Japanese PMDA approved 66 new active ingredients, including several orphan drugs, reflecting a proactive approach to rare disease treatment.

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Recent Mergers and Acquisitions:

United States: Recent M&A Activity

✅ In September 2025, Roche announced its acquisition of U.S.-based biotech firm 89bio for up to $3.5 billion. The deal aims to expand Roche's presence in treatments for liver and cardiometabolic diseases, particularly focusing on 89bio's lead candidate, pegozafermin, a late-stage therapy for metabolic dysfunction-associated steatohepatitis (MASH).

✅ In June 2025, Sanofi agreed to acquire U.S.-based Blueprint Medicines Corporation for over $9 billion. This acquisition enhances Sanofi's position in rare immunological diseases by adding Blueprint's key asset, Ayvakit/Ayvakyt, the only approved treatment for advanced and indolent systemic mastocytosis in the U.S. and EU.

✅ In May 2025, Eli Lilly announced a deal to acquire gene-editing start-up Verve Therapeutics for up to $1.3 billion. Verve is developing a CRISPR-based gene therapy targeting the PCSK9 gene, associated with high cholesterol and cardiovascular disease.

Japan: Recent M&A Activity

✅ In May 2025, Japanese pharmaceutical company Chugai Pharmaceutical announced its acquisition of U.S.-based biotech firm Vir Biotechnology for approximately $1.5 billion. The acquisition aims to strengthen Chugai's portfolio in infectious diseases, particularly focusing on Vir's monoclonal antibody therapies for hepatitis B and other viral infections.

✅ In April 2025, Takeda Pharmaceutical Company announced the acquisition of U.S.-based biotech firm GammaDelta Therapeutics for $2.3 billion. This acquisition expands Takeda's capabilities in immuno-oncology, particularly in developing gamma delta T cell therapies for various cancers.

✅ In March 2025, Daiichi Sankyo Company announced its acquisition of U.S.-based biotech firm ImmunoGen for $1.8 billion. The acquisition enhances Daiichi Sankyo's oncology pipeline, particularly focusing on ImmunoGen's antibody-drug conjugate therapies for various cancers.

Key Players:

=> Johnson & Johnson Services, Inc., AbbVie Inc., Vertex Pharmaceuticals Incorporated, AstraZeneca., Incyte., Biogen., Novartis AG, F. Hoffmann-La Roche Ltd, Pfizer Inc., and Bristol-Myers Squibb Company among others.

Growth Forecast Projected:

The Global Orphan Drugs Market is anticipated to rise at a considerable rate during the forecast period, between 2024 and 2032. In 2023, the market is growing at a steady rate, and with the rising adoption of strategies by key players, the market is expected to rise over the projected horizon.

Research Process:

Both primary and secondary data sources have been used in the global Orphan Drugs Market research report. During the research process, a wide range of industry-affecting factors are examined, including governmental regulations, market conditions, competitive levels, historical data, market situation, technological advancements, upcoming developments, in related businesses, as well as market volatility, prospects, potential barriers, and challenges.

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Key Segments:

➥ By Product Type: Small Molecules, Biologic Drugs (Gene Therapies, Monoclonal Antibodies, Others), Others

➥ By Therapy Area: Oncology, Hematology Diseases, Central Nervous System, Cardiovascular, Endocrine, Respiratory, Immunologic Conditions, Gastro-Intestinal, Musculoskeletal, Dermatology, Others

Regional Analysis for Orphan Drugs Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

Benefits of the Report:

Chapter 1: Sets the stage by outlining the report's coverage, summarizing key market segments by region, product type, and application. Presents a snapshot of market sizes, growth potential across segments, and anticipated industry evolution both short and long term.

Chapter 2: Highlights pivotal market insights and uncovers the most significant emerging trends driving change within the industry.

Chapter 3: Offers an in-depth look at the competitive landscape among Orphan Drugs producers, including revenue shares, strategic moves, and recent mergers and acquisitions.

Chapter 4: Presents comprehensive profiles of the market's key players, delving into details such as revenue, profit margins, product portfolios, and company milestones.

Chapters 5 & 6: Analyze Orphan Drugs revenue at both regional and country levels, providing quantitative breakdowns of market sizes, growth opportunities, and development prospects worldwide.

Chapter 7: Focuses on different market segments by type, examining their individual sizes and potential, guiding readers toward high-impact, untapped market areas.

Chapter 8: Explores segmentation by application, evaluating industry growth potential in various downstream markets and pinpointing promising sectors for expansion.

Chapter 9: Provides a thorough review of the industry's supply chain mapping out both upstream and downstream activities.

Chapter 10: Concludes with a summary of the report's key findings and highlights the most critical takeaways for industry stakeholders.

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FAQ

What is the current size of the Orphan Drugs Market?

A: In 2023, the Orphan Drugs Market was valued at US$ 223.76billion, reflecting its strong industry presence.

Q2: How large is the Orphan Drugs Market expected to be by 2032?

A: By 2032, industry forecasts suggest the Orphan Drugs Market will grow to around US$ 486.51 billion, demonstrating significant expansion.

Q3: What is the growth rate of the Orphan Drugs Market?

A: The market is projected to expand at a compound annual growth rate (CAGR) of 9.1% during the forecast period from 2024 to 2032.

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