The Mucopolysaccharidosis Type I (MPS I) market is expected to witness significant growth over the next decade, with a projected compound annual growth rate (CAGR) of 8% from 2025 to 2034, according to DelveInsight. In 2024, the total market size across the seven major markets (7MM)—including the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan—was valued at approximately USD 145 million. The U.S. led the market, contributing 48% of the total value, largely due to higher diagnosis rates and access to advanced therapeutic options.

The report also highlights a pipeline of promising emerging therapies poised to transform the treatment landscape for MPS I. Companies such as Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, and IMMUSOFT are developing innovative treatments like OTL-203, Lepunafusp alfa (JR-171), RGX-111, and ISP-001. These therapies aim to address the underlying causes of MPS I and improve neurological, skeletal, and systemic outcomes. Key research updates, including Orchard’s proof-of-concept study and Immusoft’s positive trial data, were presented at the 2025 WORLDSymposium.

Strategic partnerships are also shaping the future of MPS I treatment. In March 2025, REGENXBIO finalized a collaboration with Nippon Shinyaku to jointly develop and commercialize RGX-111 in the U.S. and Asia. This alliance, along with ongoing clinical research and investment in gene therapy, underscores the growing momentum in addressing the unmet needs of MPS I patients. With approximately 660 diagnosed prevalent cases in the 7MM as of 2024, the market is poised for significant clinical and commercial advancements in the coming years.

MedTech Spectrum's Summary

Market Expansion: The MPS I market is projected to grow at an 8% CAGR through 2034, indicating rising demand and investment in rare disease treatment solutions.

Therapeutic Innovation: Emerging gene and enzyme replacement therapies from leading biotech companies aim to significantly improve patient outcomes in MPS I.

Strategic Collaborations: Partnerships like REGENXBIO and Nippon Shinyaku’s alliance reflect a strong industry focus on advancing and commercializing next-generation treatments globally.