BioMarin Pharmaceutical has entered into an exclusive worldwide license agreement to gain an investigational next-generation enzyme replacement therapy that has the potential to treat Fabry disease.
The deal will see BioMarin paying $70 million upfront to GC Biopharma and Hanmi Pharm to gain worldwide rights (excluding Korea) to HM15421/GC1134A. The two companies are also eligible for additional payments of up to $405 million upon completion of development and commercial milestones as well as for tiered royalties on net sales.
The drug candidate is a long-acting, subcutaneous, clinical-stage human recombinant alpha-galactosidase A (a-Gal A) enzyme replacement therapy. Currently in a phase 1/2 clinical trial, it is the first subcutaneous enzyme replacement therapy for Fabry disease. It is the second potential treatment for the disease in BioMarin’s portfolio, thanks to the company’s acquisition of Amicus Therapeutics in December of last year.
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