GC Biopharma and Hanmi Pharmaceutical Get IND Clearance from U.S. FDA for Phase 1/2 Trial

GC Biopharma, in collaboration with Hanmi Pharmaceutical, has achieved a significant milestone in the treatment of Fabry disease with their innovative drug 'LA-GLA' (GC1134A/HM15421). This new treatment, the world's first once-monthly subcutaneous therapy for Fabry disease, recently received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for a Phase 1/2 clinical trial.

Fabry disease is a rare genetic disorder caused by a deficiency in the enzyme alpha-galactosidase A, necessary for breaking down glycolipids in lysosomes. The lack of this enzyme leads to the accumulation of glycolipids, causing cellular toxicity and inflammation, which progressively damages multiple organs. This disease can result in severe outcomes, including death if not managed properly.

Currently, the standard treatment for Fabry disease involves Enzyme Replacement Therapy (ERT), which requires intravenous administration of the enzyme every two weeks. This method is not only inconvenient but also has limitations, including the burden of frequent hospital visits and insufficient efficacy in preventing kidney disease progression.

LA-GLA aims to overcome these challenges by offering a more convenient treatment option. The once-monthly subcutaneous injection regimen of LA-GLA is designed to significantly improve patient convenience. Preclinical studies have shown that LA-GLA not only enhances kidney function but also offers superior efficacy in managing vascular disease and peripheral nerve disorders compared to existing therapies. These promising results led to its designation as an Orphan Drug by the U.S. FDA in May.

The collaborative project between GC Biopharma and Hanmi Pharmaceutical brings together the latest FDA-mandated clinical protocols and combines the specialized technical expertise of both companies. This integration of knowledge and skills has facilitated a smooth transition into the clinical phase. Both companies are committed to leveraging their extensive experience in developing treatments for Lysosomal Storage Diseases (LSD) to provide new therapeutic options for patients suffering from Fabry disease.

GC Biopharma, formerly known as Green Cross Corporation, is a leading biopharmaceutical company headquartered in Yongin, South Korea. The company has been dedicated to delivering life-saving and life-sustaining protein therapeutics and vaccines for over half a century. It is one of the global leaders in the production of plasma protein and vaccine products, consistently focusing on quality healthcare solutions.

The collaboration between GC Biopharma and Hanmi Pharmaceutical represents a significant step forward in the treatment of Fabry disease. The development of LA-GLA not only addresses the limitations of current treatment methods but also offers a more effective and convenient solution for patients. The successful IND clearance by the FDA marks the beginning of clinical trials that will assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of LA-GLA in patients with Fabry disease.

This innovative approach to treating Fabry disease has the potential to significantly improve the quality of life for patients by reducing the frequency of treatments and enhancing the overall efficacy of the therapy. The commitment of GC Biopharma and Hanmi Pharmaceutical to this project underscores their dedication to advancing medical science and providing new hope to those affected by rare genetic disorders.

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