BioMarin Pharmaceutical Enters License Agreement With GC Biopharma And Hanmi Pharm To Develop And Commercialize Its HM15421/GC1134A Investigational Long-Acting Enzyme Replacement Therapy For Fabry Disease; Company To Pay $70M Up Front, With Partners Eligible For Up To $405M In Milestones

HM15421/GC1134A is a human recombinant alpha-galactosidase A (a-Gal A) enzyme therapy currently being studied in a Phase 1/2 clinical trial in people living with Fabry disease and has the potential to be the first subcutaneous enzyme replacement therapy for Fabry disease.

"This agreement reflects our growing commitment to the Fabry community, building on the acquisition of Amicus earlier this year," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "HM15421/GC1134A has the potential to offer a new way of treating Fabry disease, and we believe it could be an important option for patients. As we continue to add to our clinical portfolio, both through internal research and external programs, HM15421/GC1134A is an excellent fit and could allow us to reach a new generation of patients with Fabry beyond those with mutations amenable to treatment with GALAFOLD."

Under the terms of the agreement, BioMarin will make an upfront payment of $70 million. GC and Hanmi are also jointly eligible to receive up to an additional $405 million upon the completion of certain development and commercial milestones, and tiered royalties.

In preclinical studies, HM15421/GC1134A demonstrated differentiated efficacy across key measures, including renal function, vascular health and peripheral nervous system outcomes. The candidate received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in 2024. Following the ongoing Phase 1/2 study, BioMarin will assume sole responsibility for all subsequent clinical development, manufacturing and commercialization of HM15421/GC1134A outside of Korea. GC and Hanmi will retain commercialization rights for HM15421/GC1134A in Korea.