From GlobeNewswire: 2025-06-20 08:30:00

The Orphan Drugs Market was valued at USD 190.87 billion in 2024 and is expected to reach USD 475.61 billion by 2032, with a CAGR of 12.11%. The U.S. market alone accounted for USD 56.25 billion in 2024, projected to hit USD 123.48 billion by 2032, driven by investments in R&D and gene-based therapies.

Regulatory support like the FDA’s Orphan Drug Act has spurred the growth of orphan drugs for rare diseases, offering advantages such as market exclusivity and expedited approval. Pharma giants are entering the market, leveraging technologies like gene therapy and monoclonal antibodies for precision medicines.

The U.S. leads in orphan drug development, with robust R&D spending and collaborations driving innovation. Government initiatives are expanding access to treatments for rare diseases, making them more widely available to patients in need.

The oncology segment dominated the orphan drugs market in 2024, with a share of 38.7%, driven by the high unmet need for treatments like glioblastoma. Biologics led in drug type, with 72% market share, offering disease-modifying results for rare diseases.

North America led the orphan drugs market in 2024, with the U.S. holding the largest share due to regulatory support and collaborations in rare disease drug development. Asia Pacific is the fastest-growing region, with Japan, China, and South Korea focusing on rare diseases and biopharma investments.

Recent developments in the orphan drugs market include FDA designations for drugs targeting neurodevelopmental disorders and ovarian cancers. Pfizer’s launch of Elrexfio for multiple myeloma under orphan status shows ongoing innovation in rare disease treatments.



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