Rare Disease Therapeutics Market Advances as Biogen Agrees to Acquire Apellis for US$5.6 Billion | Report by DataM Intelligence

Biogen's planned US$5.6 billion acquisition of Apellis, announced in March 2026, would add two commercialized medicines to its portfolio, including EMPAVELI, approved for two rare kidney diseases. The deal reflects how established drugmakers are using acquisitions to add marketed rare-disease treatments, specialist commercial teams and pipeline capabilities.

The transaction creates an opportunity to expand access to rare-disease medicines and strengthen development in nephrology. Biogen and Apellis bring complementary capabilities, while companies such as Novartis, Sanofi, Takeda and Amgen face pressure to differentiate through clinical benefit, durability, patient identification and reimbursement access. The market is also seeing momentum in gene therapy: the FDA approved Genglycos in August 2026 as the first treatment for glycogen storage disease type Ia.

According to DataM Intelligence, the global rare disease therapeutics market was valued at US$175.98 billion in 2025 and is projected to reach US$642.92 billion by 2035, at a 13.8% CAGR from 2026 to 2035.

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North America leads the market, while Asia-Pacific is expected to grow fastest as diagnosis, specialist care and access to advanced therapies expand. Enzyme replacement therapy remains the leading treatment type, accounting for 25.51% of 2025 market value; gene therapy is the fastest-growing therapy segment, supported by development of one-time treatments for genetically defined disorders.

Recent Developments

- August 28, 2026 - FDA approved Takeda's MIMRYLO for adults with polycythemia vera, a rare blood disorder. In Phase 3, 76.9% of patients achieved clinical response during weeks 20-32.

- August 18 - BioMarin agreed to acquire Alesta for US$275 million upfront plus up to US$215 million in milestones, adding oral candidate ALE1 for hypophosphatasia.

- August 12 - PTC Therapeutics won the auction for ST-920, a one-time Fabry disease gene therapy, for US$111 million upfront and up to US$100 million in regulatory milestones.

- August 6 - Tarsus agreed to buy Alkeus Pharmaceuticals, adding Phase 3 oral candidate gildeuretinol for Stargardt disease, an inherited retinal disorder without an FDA-approved treatment.

- July 9 - ARPA-H committed up to US$160 million over five years to its THRIVE program, advancing platform-based precision genetic medicines and multi-disease clinical trial models.

- June 11 - Chiesi completed its KalVista acquisition, adding EKTERLY, the first oral, on-demand treatment for hereditary angioedema attacks.

- May 18 - Neurocrine completed its Soleno acquisition, adding VYKAT XR, the first approved treatment for hyperphagia in Prader-Willi syndrome.

Regional Analysis

Japan Market
Japan's orphan designation criteria include fewer than 50,000 potential patients or inclusion on the designated intractable-disease list, alongside high medical need and a viable domestic development plan. The MHLW list reached 348 conditions in April 2026. This framework creates a route for small-population therapies, while patient identification, specialist access and local trial feasibility remain practical commercialization gates. Recent Japanese involvement is visible through Takeda's rare-disease launch activity and Shionogi's April acquisition of global ALS drug rights.

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United States Market
The U.S. Orphan Drug Act generally defines rare disease as affecting fewer than 200,000 people. North America-not the U.S. alone-represented 42.96% of global market value in 2025, according to DataM. Recent FDA approvals, the THRIVE funding program and guidance for individualized therapies show how U.S. activity combines commercial access with new evidence models for ultra-rare genetic conditions. The report does not disclose a U.S.-only market value.

Company Profiles
- Takeda: Its rare-disease portfolio spans hematology, hereditary angioedema, lysosomal storage disorders and neuroscience. MIMRYLO's U.S. approval adds a first-in-class option for polycythemia vera.

- BioMarin: The company focuses on genetically defined conditions and reports nine commercial therapies. Its Alesta deal adds ALE1, an oral clinical-stage candidate for hypophosphatasia.

- PTC Therapeutics: PTC markets rare-disease treatments including Sephience, Emflaza and Translarna. The ST-920 acquisition expands its gene-therapy pipeline in Fabry disease.

- Chiesi: Its Global Rare Diseases unit develops and commercializes rare and ultra-rare therapies. The KalVista acquisition added EKTERLY, approved in the U.S., Japan and other markets.

Market Segmentation

By Therapy Type
Enzyme replacement therapy led the market in 2025 with 25.51%, equal to US$44.89 billion. Established use in lysosomal storage and inherited metabolic disorders supports its revenue base.

By Modality
Gene therapy is forecast to be the fastest-growing therapy segment, at an indicative 26% CAGR through 2035. One-time treatment potential is attractive, though durability, safety monitoring, reimbursement and access to eligible patients shape uptake.

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