Hanmi Pharm and GC Biopharma said LA-GLA (code name: HM15421/GC1134A), a Fabry disease drug candidate being co-developed by the two companies, has won orphan drug designation (ODD) from the U.S. FDA.

The FDA's orphan drug system is designed to facilitate the development and approval of drugs to treat rare diseases that affect 200,000 or fewer people in the U.S. Drugs granted orphan drug designation receive tax credits for clinical development costs, reduced fees for reviewing applications, and exclusive marketing in the U.S. for seven years.

LA-GLA is a next-generation, long-acting enzyme replacement therapy (ERT) for Fabry disease, a rare lysosomal storage disorder caused by a deficiency in the enzyme alpha-galactosidase A.

This enzyme deficiency leads to the accumulation of glycosphingolipids in cells, causing cellular toxicity, inflammation, and progressive damage to multiple organs, ultimately resulting in death if untreated.

The current standard treatment for Fabry disease involves biweekly intravenous enzyme replacement therapy, which, while effective, poses several challenges, including frequent hospital visits, lengthy infusion times, and limited efficacy in slowing the progression of renal disease.

However, the two companies believe LA-GLA can represent a significant advancement with its once-monthly subcutaneous injection regimen, addressing many of the limitations of first-generation therapies.

At the WORLD Symposium 2024, held in San Diego last February, Hanmi presented two pivotal studies demonstrating LA-GLA's superior efficacy in improving kidney function, vascular health, and peripheral neuropathy compared to existing treatments.

Building on these results, Hanmi and GC are preparing to file a global Investigational New Drug (IND) application to advance LA-GLA into clinical trials.