US-based BioMarin Pharmaceutical Inc. has entered into an exclusive worldwide license agreement with South Korea-based GC Biopharma Corp. and Hanmi Pharm Co., to develop and commercialise HM15421/GC1134A, an investigational long-acting enzyme replacement therapy for Fabry disease.
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.
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.
Fabry disease is a lysosomal storage disorder caused by inherited genetic changes in the GLA gene, which hampers activity of the enzyme alpha-galactosidase A (a-Gal A).
