BioMarin Secures Global License for Innovative Treatment for Fabry Disease

BioMarin Enters Exclusive License to Develop Groundbreaking Fabry Disease Treatment

BioMarin Pharmaceutical Inc., a leading company in the biotechnology sector, has taken a significant step in improving treatment options for Fabry disease by signing an exclusive worldwide license agreement with GC Biopharma Corp. and Hanmi Pharm Co., Ltd. This strategic partnership will pave the way for the development and commercialization of a promising new treatment option, HM15421/GC1134A, aimed at addressing this rare genetic disorder that leads to severe health complications.

What is Fabry Disease?

Fabry disease is a lysosomal storage disorder caused by genetic mutations in the GLA gene, which disrupts the production of the enzyme alpha-galactosidase A (a-Gal A). This deficiency results in the harmful accumulation of globotriaosylceramide (GL-3) in various organs, leading to an array of symptoms including pain, kidney failure, and an increased risk of stroke and heart attacks. With symptoms that can be severe and varied among individuals, there is a pressing need for effective treatments.

The New Therapy: HM15421/GC1134A

HM15421/GC1134A represents a novel approach in enzyme replacement therapy, designed to be administered subcutaneously. It boasts a longer action duration compared to existing treatments and is currently undergoing a Phase 1/2 clinical trial. Early preclinical studies show promising results across multiple health indicators, including kidney function, vascular health, and outcomes affecting the peripheral nervous system.

Under the terms of the agreement, BioMarin will pay an upfront fee of $70 million and is poised to make additional payments totaling up to $405 million based on the achievement of specific development milestones. Furthermore, BioMarin will also be entitled to tiered royalties on the net sales of the therapy once it hits the market outside Korea, where GC Biopharma and Hanmi hold the commercialization rights.

Commitment to the Fabry Community

Alexander Hardy, BioMarin's President and CEO, expressed enthusiasm about the agreement, emphasizing its alignment with the company's commitment to the Fabry community. The acquisition of this new potential treatment complements BioMarin's recent acquisition of Amicus Therapeutics, enhancing its portfolio aimed at addressing the needs of patients with rare diseases.

Hardy noted, "HM15421/GC1134A has the potential to provide a new treatment paradigm for Fabry disease, and we are excited to continue expanding our clinical pipeline with options that can make a real difference in patients' lives."

The Path Ahead

Following the ongoing clinical studies, BioMarin aims to take complete control of the clinical development and commercialization process for HM15421/GC1134A outside of Korea. This therapy’s successful commercialization could transform patient care for those battling Fabry disease and present a valuable option for individuals not currently suitable for existing treatments like GALAFOLD.

As BioMarin moves forward with this promising therapy, the company remains committed to its mission of providing innovative solutions for genetically defined conditions, demonstrating its dedication to research-driven approaches and patient-centric care.

For more information about BioMarin and its therapies, visit www.biomarin.com.