BioMarin Pharmaceutical has announced a major strategic expansion into the rare disease space, confirming an exclusive, worldwide license agreement with South Korean pharmaceutical leaders GC Biopharma and Hanmi Pharm. The partnership centers on the development and commercialization of HM15421/GC1134A, a promising, long-acting enzyme replacement therapy (ERT) designed to treat Fabry disease.
Fabry disease is a debilitating lysosomal storage disorder triggered by inherited genetic changes in the GLA gene. These mutations significantly diminish the activity of the enzyme alpha-galactosidase A (a-Gal A), causing a harmful accumulation of globotriaosylceramide (GL-3) within cells. This buildup leads to severe, life-altering symptoms, including chronic pain, kidney failure, and a significantly heightened risk of heart attack and stroke.
The investigational drug HM15421/GC1134A is currently being evaluated in a Phase I/II clinical trial and holds the potential to be a first-in-class subcutaneous enzyme replacement therapy for the condition. Preclinical data for the treatment have been highly encouraging, demonstrating consistent efficacy across critical health markers, including improved renal function, vascular health, and outcomes related to the peripheral nervous system.
Under the terms of the agreement, the global responsibilities will be split. BioMarin will take the lead on the clinical development, manufacturing, and commercialization of the therapy for all markets outside of South Korea. In a notable caveat to the deal, GC Biopharma and Hanmi Pharm will retain full commercialization rights within the Korean market, ensuring their continued participation in the drug’s lifecycle.
The financial scope of the partnership is significant, reflecting the high stakes of the rare disease market. BioMarin is set to make an upfront payment of $70 million to its partners. Furthermore, GC and Hanmi are eligible to receive up to $405 million in additional payments contingent upon the successful completion of specific development and regulatory milestones.
BioMarin President and CEO Alexander Hardy emphasized that this collaboration aligns with the company’s broader strategy to cement its leadership in the Fabry community, a commitment highlighted by the company’s recent $4.8 billion acquisition of Amicus Therapeutics.
“HM15421/GC1134A has the potential to offer a new way of treating Fabry disease,” Hardy stated. “We believe it could be an important option for patients. As we continue to add to our clinical portfolio… HM15421/GC1134A is an excellent fit. [It] could allow us to reach a new generation of patients with Fabry beyond those with mutations amenable to treatment.”
The therapeutic candidate has already gained traction with regulators, having been granted Orphan Drug Designation by the U.S. Food and Drug Administration earlier this year. With this new partnership, all three companies aim to accelerate the path to market for a therapy that could fundamentally change the standard of care for patients suffering from Fabry disease.
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