GC Biopharma, Hanmi Pharmaceutical Out-License Fabry Drug for Up to $475M

KRW 93.5 billion upfront, milestones of up to KRW 540 billion
Deal comes six years after co-development began; South Korean rights retained

GC Biopharma and Hanmi Pharmaceutical have out-licensed a Fabry disease drug candidate they have been developing together to U.S. rare-disease specialist BioMarin for up to $475 million (approximately KRW 634.6 billion). The deal marks a milestone achieved six years after the two companies began co-developing the drug.

BioMarin said on October 29 local time that it had signed a global exclusive license agreement with GC Biopharma and Hanmi Pharmaceutical for the Fabry disease treatment candidate HM15421/GC1134A. The upfront payment is $70 million (approximately KRW 93.5 billion), with development and commercialization milestones totaling up to $405 million (approximately KRW 541.1 billion). BioMarin will also pay royalties based on net sales after commercialization.

BioMarin will have the right to develop and sell the candidate worldwide, excluding South Korea. BioMarin will solely handle clinical trials following the ongoing Phase 1/2 trial, manufacturing, and commercialization outside South Korea. GC Biopharma and Hanmi Pharmaceutical will retain the domestic sales rights.

Fabry disease is a rare genetic disorder in which a genetic mutation prevents an enzyme that breaks down glycolipids from functioning properly. As a result, fatty substances accumulate in organs including the kidneys, heart, and nerves, causing damage. The current standard treatment is enzyme replacement therapy, which supplies the missing enzyme from outside the body. However, it has been burdensome for patients, who must visit a hospital every two weeks to receive intravenous infusions lasting several hours.

HM15421/GC1134A is being developed to keep the enzyme in the body longer, extend the intervals between doses, and allow administration by subcutaneous rather than intravenous injection. The goal is to administer it once a month.

The two companies signed an agreement in 2020 to co-develop treatments for lysosomal storage disorders. In 2024, the U.S. Food and Drug Administration (FDA) approved the Phase 1/2 trial and designated the candidate an orphan drug. In South Korea, the companies received approval for a Phase 1/2 trial in January 2025 and began dosing the first patient in May.

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