[E-Daily Reporter KIM SAE-MI ] Green Cross Corporation(006280)and HanmiPharm(128940)have licensed a Fabry disease drug candidate they jointly developed to BioMarin, a U.S.-based pharmaceutical company specializing in rare diseases, in a deal worth up to $475 million (approximately 634.6 billion won). This achievement comes approximately six years after the two companies began their joint development in 2020.

According to the biotech industry on the 30th, BioMarin announced on the 29th (local time) that it had signed a global exclusive licensing agreement with GC Biopharma Corp. and HanmiPharm for the Fabry disease drug candidate “HM15421/GC1134A (LA-GLA).”

The total value of the agreement is $475 million (634.6 billion won), comprising an upfront payment of $70 million (approximately 93.5 billion won) and milestone payments of up to $405 million (541.1 billion won) based on development and commercialization milestones. If the product is successfully commercialized in the future, separate tiered royalties linked to net sales will also be paid. It is reported that the two companies will split the technology fees resulting from this agreement 50-50.

Through this agreement, BioMarin has secured the exclusive rights to develop and commercialize HM15421/GC1134A worldwide, excluding South Korea. The company plans to take sole responsibility for subsequent clinical development, production, and international commercialization following the ongoing Phase 1/2 clinical trial. GC Biopharma Corp. and HanmiPharm will retain the commercialization rights in South Korea.

HM15421/GC1134A is a long-acting enzyme replacement therapy (ERT) designed to replenish the alpha-galactosidase A (α-Gal A) enzyme, which is deficient in patients with Fabry disease. Unlike existing intravenous administration methods, it is being developed for subcutaneous injection. The two companies aim to develop a treatment that can be administered subcutaneously once a month.

Fabry disease is a rare genetic disorder caused by a mutation in the GLA gene, which reduces the activity of a specific enzyme and leads to the accumulation of glycolipids in the body. It can cause damage to the kidneys, heart, and peripheral nervous system. Existing enzyme replacement therapy (ERT) treatments have the drawback of requiring intravenous injections primarily every two weeks.

GC Biopharma Corp. and HanmiPharm signed a joint development agreement in 2020 to develop next-generation drugs for the treatment of lysosomal storage disorders. Subsequently, in 2024, they received approval from the U.S. Food and Drug Administration (FDA) for their Phase 1/2 clinical trial protocol, and in January of last year, they also obtained approval for the Phase 1/2 clinical trial in South Korea. In May of the same year, they began administering the drug to the first patients.

HM15421/GC1134A was designated as an FDA Orphan Drug in 2024. According to BioMarin, nonclinical studies confirmed its differentiated efficacy in indicators related to kidney function, vascular health, and the peripheral nervous system.

Through this technology acquisition, BioMarin plans to expand its portfolio of Fabry disease treatments. Previously, in April, BioMarin acquired U.S.-based Amicure Therapeutics for approximately $4.8 billion (about 6.4128 trillion won), securing the oral Fabry disease treatment “Galafold” (active ingredient: migalastat).

Galafold is a treatment for Fabry disease patients with specific genetic mutations. BioMarin expects that if HM15421/GC1134A is successfully commercialized in the future, it will expand treatment options to include patients with genetic mutations for whom Galafold is not suitable.

Alexander Hardy, CEO of BioMarin, stated, "This agreement, following the acquisition of Amicus earlier this year, demonstrates our commitment to developing treatments for patients with Fabry disease," adding, "HM15421/GC1134A has the potential to serve as a new treatment modality for Fabry disease and could broaden treatment opportunities for patients with genetic mutations that make them unsuitable for Galafold therapy."