GC Biopharma and Hanmi Pharmaceutical have announced that their co-developed Fabry disease treatment, LA-GLA (GC1134A/HM15421), has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for a Phase 1/2 clinical trial. This milestone marks a significant step forward in developing a more convenient and potentially more effective therapy for Fabry disease patients.
LA-GLA is designed as a novel enzyme replacement therapy administered via a once-monthly subcutaneous injection. Current treatments for Fabry disease involve Enzyme Replacement Therapy (ERT), which requires patients to undergo intravenous infusions every two weeks. This frequent hospital visits can be burdensome and inconvenient for patients.
Addressing Limitations of Current Fabry Disease Treatments
Existing ERT treatments, while effective to some extent, have limitations, including the burden of prolonged intravenous therapy and insufficient efficacy in preventing the progression of kidney disease. LA-GLA aims to address these limitations by offering a more convenient administration route and potentially improved efficacy.
Preclinical Evidence and Orphan Drug Designation
Preclinical studies of LA-GLA have demonstrated promising results, showing improvements in kidney function and superior efficacy in managing vascular disease and peripheral nerve disorders compared to existing therapies. These positive findings led to the U.S. FDA granting LA-GLA Orphan Drug Designation (ODD) in May.
Clinical Trial Objectives
The Phase 1/2 clinical trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of LA-GLA in patients with Fabry disease. This trial is crucial for determining the optimal dosing regimen and assessing the overall clinical benefit of LA-GLA.
Fabry Disease: A Rare Genetic Disorder
Fabry disease is a rare genetic disorder linked to the X chromosome and classified as a type of Lysosomal Storage Disease (LSD). It results from a deficiency in the enzyme alpha-galactosidase A, which is essential for breaking down glycolipids in lysosomes. The accumulation of glycolipids due to this deficiency leads to cellular toxicity and inflammatory responses, progressively damaging multiple organs and potentially leading to severe outcomes, including death.
GC Biopharma and Hanmi Pharmaceutical stated that this collaboration integrates the latest FDA-mandated clinical protocols and leverages the specialized technical expertise of both companies, enabling a rapid transition into the clinical phase. They further added that they will do their utmost to create new treatment options to patients suffering from Fabry disease, based on their expertise and knowledge of developing new treatments for Lysosomal Storage Disease (LSD).
