BioMarin signs up to $475 million licensing deal for new Fabry disease enzyme therapy

BioMarin announced a licensing agreement to acquire rights to a long‑acting enzyme replacement therapy candidate for Fabry disease, paying $70 million upfront and up to $405 million in development and commercial milestones.

The candidate, identified as HM15421/GC1134A, is being developed by South Korea’s GC Biopharma and Hanmi Pharm, which have already begun a phase 1/2 first‑in‑human study enrolling patients in the United States, Argentina and South Korea.

The deal expands BioMarin’s Fabry franchise, which already includes the oral therapy Galafold acquired through its $4.8 billion purchase of Amicus Therapeutics and generated $521.7 million in net sales in 2025.

Under the agreement BioMarin will hold worldwide commercial rights except in South Korea, where the original partners retain rights, and will pay tiered royalties on future sales.