Hanmi Pharm and GC Biopharma are entering global phase 1/2 global clinical trials for LA-GLA (HM15421/GC1134A), a once-monthly subcutaneous treatment for Fabry disease. 

The trial, approved by the Ministry of Food and Drug Safety last Thursday and by the U.S. FDA in August 2024, will evaluate the treatment’s safety, tolerability, and efficacy.

Fabry disease, a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A, leads to toxic glycosphingolipid accumulation in cells, resulting in organ damage. Current therapies require biweekly intravenous infusions and have limitations in addressing kidney disease, a common complication.

Hanmi’s LA-GLA is designed as a next-generation extended enzyme replacement therapy, offering improved durability, safety, and convenience, according to the company. The treatment received Orphan Drug Designation from the FDA in May 2024. 

Hanmi Pharm plans to present three preclinical studies of LA-GLA at the WORLDSymposium 2025 in San Diego next month, highlighting improved outcomes in kidney function, vascular health, and peripheral nerve disorders compared to existing therapies.