HM15421

HM15421 (also known as GC1134A or LA-GLA) is a long-acting recombinant alpha-galactosidase enzyme replacement therapy developed for the treatment of Fabry disease. Fabry disease is a rare genetic disorder caused by deficiency of the enzyme alpha-galactosidase A, leading to accumulation of glycolipids and progressive organ damage. Unlike current standard therapies that require intravenous infusions every two weeks, HM15421 is designed for once-monthly subcutaneous administration to improve patient convenience and adherence. Preclinical studies have shown improved kidney function and efficacy in managing vascular and peripheral nerve complications compared to existing treatments. The drug has received Orphan Drug Designation from the US FDA and entered Phase 1/2 clinical trials in 2025[2][3][7][8].

Other names

Long-acting alpha-galactosidaseLong-acting GLA