Orphan Drug Market to Reach USD 621.85 Billion by 2034, Driven by Rising Rare Disease Incidence and Innovation

The global orphan drug market size stood at US$ 216.66 billion in 2025, grew to US$ billion in 2026, and is forecast to reach US$ 621.85 billion by 2034, expanding at a CAGR of 12.24% from 2026 to 2035.

Ottawa, Nov. 10, 2025 (GLOBE NEWSWIRE) — The global orphan drug market size was valued at USD 193.05 billion in 2024 and is predicted to hit around USD 621.85 billion by 2034, rising at a 12.24% CAGR, a study published by Towards Healthcare a sister firm of Precedence Research. The global orphan drug market is driven by the increasing incidence of rare diseases and growing innovations.

The Complete Study is Now Available for Immediate Access | Download the Sample Pages of this Report @ https://www.towardshealthcare.com/download-sample/5543

Key Takeaways

• North America held a major revenue share by 47% of the market in 2024.
• Europe is expected to witness the fastest growth in the orphan drug market during the forecast period.
• By drug type, the biologicals segment held a major revenue share of the market in 2024.
• By drug type, the non-biologicals segment is expected to witness significant growth in the market during the forecast period
• By therapy, the oncology segment held a major revenue share of the market in 2024.
• By therapy, the neurology segment is expected to witness the fastest growth in the market during the forecast period
• By distribution channel, the hospital pharmacies segment held a major revenue share of the market in 2024.
• By distribution channel, the online pharmacies segment is expected to witness significant growth in the market during the forecast period.

What is the Orphan Drug Market?

The orphan drugs market is driven by the growing prevalence of rare diseases, significant technological advancements in medicine, and supportive government regulations. The orphan drugs refer to the medications developed for the treatment of rare diseases that affect only a small percentage of the population. The rare diseases, such as genetic disorders, metabolic disorders, rare autoimmune conditions, rare cancers, and rare neurological conditions, require orphan drugs for their treatment.

You can place an order or ask any questions, please feel free to contact us at [email protected]

Key Metrics and Overview

What are the Major Growth Drivers in the Orphan Drug Market?

Growing government initiatives are the major growth driver in the market, which is supporting the approval and adoption of orphan drugs. They are offering tax credits and funding for clinical trials and their development, which is accelerating their innovations and encouraging companies to invest in the development of treatments for rare diseases. Additionally, advancements in biotechnology, personalized medicine, R&D investments, technological advancements, and growing awareness are other market drivers.

What are the Key Drifts in the Orphan Drug Market?

The market has been expanding due to the growing funding and acquisitions to develop and launch different types of orphan drugs.

• In November 2025, a $141 million (€122 million) Series B financing round was announced to have successfully closed by AAVantgarde Bio. This funding will be used to support the completion of the clinical PoC of its AAVB-039 CELESTE in patients with Stargardt disease, which was granted Orphan Drug Designation (ODD) by the U.S. FDA.
• In May 2025, a total of €132,000,000 in funding was secured by Azafaros B.V. to accelerate the clinical development and regulatory approval of nizubaglustat, which is a novel small molecule therapy targeting lysosomal storage diseases with no effective treatments.
• In January 2025, a front-loaded acquisition agreement was signed by GSK for the acquisition of IDRx and its rare cancer drug candidate, where $1 billion and $150 million will be offered by GSK for upfront payment and regulatory approval, respectively.

What is the Significant Challenge in the Orphan Drug Market?

High cost acts as the major challenge in the market. The cost associated with the orphan drugs is high, as they require specialized manufacturing, complex R&D, and are used by a small patient population, which limits access by patients and healthcare systems in developing countries. Moreover, limited patient population, stringent guidelines, lack of accurate diagnostics, limited infrastructure, and reimbursement challenges are other market restraints.

Become a valued research partner with us – https://www.towardshealthcare.com/schedule-meeting

Regional Analysis

Why did North America Dominate the Orphan Drug Market in 2024?

In 2024, North America captured the biggest revenue share in the market, due to a robust regulatory framework that provides incentives to the industries for developing drugs for rare diseases. At the same time, the well-developed healthcare system provides reimbursement policies to orphan drugs to make them affordable and accessible to the patients, where growing patient awareness has enhanced their use. Additionally, the companies are supported by investments and funding, which have increased their R&D as well as the development of personalized medications, which contributed to the market growth.

What Made the Asia Pacific Grow Fastest in the Orphan Drug Market in 2024?

Asia Pacific is expected to host the fastest growth in the market during the forecast period, due to the presence of a large population, which increases the risk of incidences of rare diseases, driving the demand for orphan drugs. At the same time, the expanding healthcare sector is increasing the adoption of advanced diagnostics, accelerating their early diagnosis and treatment. Additionally, the government policies are supporting the adoption, development, and innovations of orphan drugs, where the industries are also accelerating their manufacturing, leading to new collaborations, launches, and approvals, promoting the market growth.

Get the latest insights on life science industry segmentation with our Annual Membership: https://www.towardshealthcare.com/get-an-annual-membership

Segmental Insights

By Drug Analysis

By drug type, the biologicals segment held the largest share of the market in 2024, due to their high specificity. This, in turn, helped in providing high efficacy and low side effects, which increased their use. At the same time, due to unmet needs for various rare diseases, they are the preferred treatment option.

By drug type, the non-biologicals segment is expected to show a lucrative growth rate in the market during the predicted time, due to their oral route of administration, which is increasing patient convenience. Moreover, their simple manufacturing and wide range of applications are increasing their use. Similarly, their generic formulation are enhancing their adoption rates.

By Therapy Analysis

By therapy, the oncology segment held the dominating share of the market in 2024, driven by the growing incidence. At the same time, the limited effective treatment options enhanced the demand for orphan drugs. Additionally, the growth in the R&D backed by investments contributed to their enhanced innovations.

By therapy, the neurology segment is expected to show the highest growth in the market during the predicted time, due to unmet medical needs. The growing innovations in genetics for target-specific treatment are enhancing the development of orphan drugs for these diseases. Furthermore, the regulatory support and funding are also encouraging their advancement and approvals.

By Distribution Channel Analysis

By distribution channel, the hospital pharmacies segment led the market in 2024, as they offered various complex treatments. Moreover, close patient monitoring and specialized handling of orphan drugs enhanced the patient outcomes. Additionally, the presence of reimbursement and insurance policies also attracted the patients.

By distribution channel, the online pharmacies segment is expected to show a lucrative growth in the market during the upcoming years, driven by their widespread availability, which enhances access across remote areas. Furthermore, their home deliveries and discounts are enhancing patient convenience. The growing online counselling platforms and disease management tools are also attracting patients.

Browse More Insights of Towards Healthcare:

The global clinical nutrition market size is estimated at US$ 78.15 billion in 2025, increased to US$ 84.48 billion in 2026, and is expected to reach around US$ 170.12 billion by 2035. The market is growing at a CAGR of 8.1% between 2026 and 2035.

The global head & neck cancer therapeutics market size is calculated at US$ 2.65 billion in 2025, grew to US$ 2.97 billion in 2026, and is projected to reach around US$ 8.14 billion by 2035. The market is expanding at a CAGR of 11.87% between 2026 and 2035.

The global microbiology culture market size is calculated at US$ 9.38 billion in 2025, grew to US$ 9.93 billion in 2026, and is projected to reach around US$ 16.55 billion by 2035. The market is expanding at a CAGR of 5.85% between 2026 and 2035.

The global intraoperative radiation therapy market size is calculated at USD 69.6 million in 2025, grew to USD 74.09 million in 2026, and is projected to reach around USD 130.18 million by 2035. The market is expected to expand at a CAGR of 6.45% from 2026 to 2035.

The global postpartum health supplements market size is calculated at US$ 1.52 billion in 2025, grew to US$ 1.66 billion in 2026, and is projected to reach around US$ 3.66 billion by 2035. The market is expanding at a CAGR of 9.14% between 2026 and 2035.

The global psoriatic arthritis treatment market size is calculated at USD 10.91 billion in 2025, grew to USD 11.94 billion in 2026, and is projected to reach around USD 26.76 billion by 2035. The market is expanding at a CAGR of 9.44% between 2026 and 2035.

The global NUT midline carcinoma treatment market size is estimated at US$ 23.92 billion in 2025, grew to US$ 26.59 billion in 2026, and is expected to reach approximately US$ 68.89 billion by 2035. The market is expanding at a CAGR of 11.15% from 2026 to 2035.

The diabetes management supplements market size stood at US$ 7.17 billion in 2025, grew to US$ 8.38 billion in 2026, and is forecast to reach US$ 17.7 billion by 2035, expanding at a CAGR of 8.65% from 2026 to 2035.

The global drug repurposing market size is calculated at US$ 636.95 million in 2025, grew to US$ 730.96 million in 2026, and is projected to reach around US$ 2506.64 million by 2035. The market is expanding at a CAGR of 14.76% between 2026 and 2035.

The global sciatica treatment market size is calculated at USD 796 million in 2025, grew to USD 827.37 million in 2026, and is projected to reach around USD 1171.76 million by 2035. The market is expected to expand at a CAGR of 3.94% between 2026 and 2035.

Recent Developments in the Orphan Drug Market

• In April 2025, rilzabrutinib targeting reversible Bruton’s tyrosine kinase (BTK) inhibitor, for two rare diseases, IgG4-related disease (IgG4-RD) and warm autoimmune hemolytic anemia (wAIHA), was granted orphan drug designation by the U.S. FDA.
• In November 2025, M2T-CD33, developed by Leukogene Therapeutics Inc., for the treatment of Acute Myeloid Leukemia, Orphan Drug Designation was granted by the U.S. FDA.
• In October 2025, for the treatment of idiopathic pulmonary fibrosis (IPF), buloxibutid, developed by Vicore Pharma Holding AB, was granted Orphan Drug designation by Japan’s Ministry of Health, Labor and Welfare (MHLW).

Orphan Drug Market Key Players List

• Merk & Co., Inc.
• F. Hoffmann-La Roche Ltd.
• Novartis AG
• Celgene Corporation
• Alexion Pharmaceuticals Inc.
• AstraZeneca
• Sanofi
• Amgen Inc.
• Takeda Pharmaceutical Company Limited
• Eli Lilly and Company

Segments Covered in The Report

By Drug Type

• Biologicals
• Non-Biologicals

By Therapy

• Oncology
  • Ovarian cancer
  • Multiple Myeloma
  • Pancreatic Cancer
  • Renal Cell Carcinoma
  • Others
• Hematology
  • Hereditary Angioedema
  • Hemophilia
  • Others
• Neurology
  • Alzheimer’s Disease
  • Huntington’s Disease
  • Duchenne Muscular Dystrophy
  • Others
• Infectious Diseases
• Metabolic Disorders
  • Gaucher Disease
  • Hypoparathyroidism
  • Hunter Syndrome
  • Fabry Disease
  • Others
• Endocrinology
• Immunology
• Others

By Distribution Channel

• Hospital Pharmacies
• Retail Pharmacies
• Online Pharmacies
• Others

By Region

North America 

• U.S. 
• Canada 
• Mexico 
• Rest of North America 

 South America 

• Brazil 
• Argentina 
• Rest of South America 

Europe 

• Western Europe  
  • Germany 
  • Italy 
  • France 
  • Netherlands 
  • Spain 
  • Portugal 
  • Belgium 
  • Ireland 
  • UK 
  • Iceland 
  • Switzerland 
  • Poland 
  • Rest of Western Europe 
• Eastern Europe  

• Austria 
• Russia & Belarus 
• Türkiye 
• Albania 
• Rest of Eastern Europe 

Asia Pacific

• China 
• Taiwan 
• India 
• Japan 
• Australia and New Zealand,  
• ASEAN Countries (Singapore, Malaysia) 
• South Korea 
• Rest of APAC 

 MEA 

• GCC Countries 
• Saudi Arabia 
• United Arab Emirates (UAE) 
• Qatar 
• Kuwait 
• Oman 
• Bahrain 
• South Africa 
• Egypt 
• Rest of MEA 

Immediate Delivery Available | Buy This Premium Research @ https://www.towardshealthcare.com/checkout/5543

Access our exclusive, data-rich dashboard dedicated to the healthcare market – built specifically for decision-makers, strategists, and industry leaders. The dashboard features comprehensive statistical data, segment-wise market breakdowns, regional performance shares, detailed company profiles, annual updates, and much more. From market sizing to competitive intelligence, this powerful tool is one-stop solution to your gateway.

Access the Dashboard: https://www.towardshealthcare.com/access-dashboard

About Us

Towards Healthcare is a leading global provider of technological solutions, clinical research services, and advanced analytics, with a strong emphasis on life science research. Dedicated to advancing innovation in the life sciences sector, we build strategic partnerships that generate actionable insights and transformative breakthroughs. As a global strategy consulting firm, we empower life science leaders to gain a competitive edge, drive research excellence, and accelerate sustainable growth.

You can place an order or ask any questions, please feel free to contact us at [email protected]

Europe Region: +44 778 256 0738

North America Region: +1 8044 4193 44

APAC Region: +91 9356 9282 04

Web: https://www.towardshealthcare.com

Our Trusted Data Partners

Precedence Research | Statifacts | Towards Packaging | Towards Automotive | Towards Food and Beverages | Towards Chemical and Materials | Towards Consumer Goods | Towards Dental | Towards EV Solutions | Nova One Advisor | Healthcare Webwire | Packaging Webwire | Automotive Webwire | Nutraceuticals Func Foods | Onco Quant | Sustainability Quant | Specialty Chemicals Analytics

Find us on social platforms: LinkedIn | Twitter | Instagram | Medium | Pinterest

Disclaimer: The above press release comes to you under an arrangement with GlobeNewswire. DailyIndiaNews.com takes no editorial responsibility for the same.