Hanmi and GC Biopharma Begin Fabry Disease Trials in Korea

Hanmi Pharmaceutical and GC Biopharma are set to begin clinical trials in Korea for their Fabry disease treatment, jointly developed as the world’s first once-monthly subcutaneous injection.

The two companies announced on the 13th that they received approval from the Korean Ministry of Food and Drug Safety (MFDS) on the 9th for their investigational new drug (IND) application to conduct Phase 1 and Phase 2 clinical trials for LA-GLA (HM15421/GC1134A).

This approval follows the U.S. FDA’s IND approval last August for the same trials, accelerating multinational clinical development. The trials will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of LA-GLA in patients with Fabry disease.

Fabry disease is a rare, progressive lysosomal storage disorder (LSD) inherited via the X chromosome. It occurs due to a deficiency of the enzyme alpha-galactosidase A, responsible for breaking down glycolipids in lysosomes, cellular organelles that remove waste. The accumulation of undigested glycolipids leads to cell toxicity and inflammation, progressively damaging various organs and potentially resulting in death.

Currently, most Fabry patients undergo enzyme replacement therapy (ERT) with recombinant enzymes administered intravenously. These first-generation treatments require biweekly hospital visits for lengthy infusions, posing significant inconvenience and treatment burden. Moreover, they show limited efficacy in preventing progressive kidney disease.

LA-GLA, a next-generation long-acting ERT, is designed to address these limitations. If commercialized, it is expected to significantly improve therapeutic durability, safety, and patient convenience, thereby reducing the burden of treatment. Recognizing its potential, the U.S. FDA designated LA-GLA as an orphan drug in May last year.

Next month, Hanmi Pharmaceutical will present three preclinical studies on LA-GLA at the World Symposium 2025 in San Diego. These studies demonstrate superior efficacy in improving kidney function, vascular complications, and peripheral neuropathy compared to existing treatments.

A representative from Hanmi Pharmaceutical and GC Biopharma commented, “The Phase 1/2 clinical trials represent a critical step in verifying the potential of this next-generation rare disease treatment. We are optimistic about the outcomes and aim to overcome the limitations of current ERTs through innovative therapies.”