GC Biopharma, Hanmi Pharmaceutical out-license Fabry disease treatment candidate for 630 billion won

[Financial News] GC Biopharma Corp. and Hanmi Pharmaceutical have out-licensed a next-generation Fabry disease treatment candidate they are jointly developing to a global rare-disease specialist. As a drug candidate secured through joint R&D between Korean pharmaceutical companies enters the global commercialization stage, the deal is also drawing attention as a result of open innovation.
GC Biopharma Corp. and Hanmi Pharmaceutical said on the 30th that they had signed an exclusive license agreement with U.S.-based BioMarin Pharmaceutical for their jointly developed Fabry disease treatment candidate, “LA-GLA (HM15421·GC1134A).”
The agreement is worth a total of $475 million (about 634.7 billion won), including an upfront payment of $70 million (about 93.5 billion won) and milestone payments of up to $405 million (about 541.2 billion won) tied to development and commercialization stages. The companies will also receive separate royalties after the product launches.
Under the agreement, BioMarin will handle development and commercialization worldwide, excluding South Korea. In South Korea, GC Biopharma Corp. and Hanmi Pharmaceutical will lead the regulatory approval process and commercialization.
LA-GLA is a next-generation, long-acting enzyme replacement therapy (ERT) candidate targeting Fabry disease, a type of lysosomal storage disorder. While existing Fabry disease ERTs are typically administered intravenously every two weeks, LA-GLA was designed for subcutaneous (SC) administration by improving the drug’s half-life and stability. Its key differentiator is that it makes administration more convenient and reduces the treatment burden on patients.
Preclinical studies confirmed efficacy on key indicators, including kidney function and measures related to the blood vessels and peripheral nervous system. The candidate received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) in 2024, and is currently in global Phase 1/2 clinical trials.
The candidate was developed by combining the companies’ core technologies. It uses Hanmi Pharmaceutical’s proprietary long-acting drug platform technology, supplemented by GC Biopharma Corp.’s experience in enzyme replacement therapy manufacturing and clinical development, accumulated through the development and commercialization of Hunterase. The companies have pursued global clinical development through joint research, and under the agreement, BioMarin will lead global development and commercialization.
BioMarin, the partner company, specializes in developing treatments for rare genetic diseases and commercializing them globally. It recently strengthened its business capabilities in Fabry disease treatments by acquiring Amicus Therapeutics. Drawing on its development and commercialization experience in rare diseases, BioMarin is expected to accelerate the global development of LA-GLA.
Lim Joo-hyun, vice chairman of Hanmi Pharmaceutical Group, said, “This is a meaningful achievement for open innovation, as joint research between Korean pharmaceutical companies has enhanced the prospects for developing innovative drugs for the global market,” adding, “We will accelerate global development while also providing new treatment options to patients in South Korea.”
Huh Eun-cheol, CEO of GC Biopharma Corp., said, “This is a meaningful result of the close R&D collaboration between the two companies,” adding, “We will expand global partnering and commercialization based on our competitive pipeline.”
[email protected] Jung Sang-hee Reporter