FDA Approves IND for GC/Novel Pharma's Sanfilippo Syndrome Treatment

GC Biopharma and Novel Pharma have achieved a significant milestone with the FDA's Investigational New Drug (IND) approval for their treatment 'GC1130A' aimed at addressing MPS IIIA. This approval marks a crucial step forward, paving the way for multinational clinical trials that will commence this year across the United States, South Korea, and Japan. The primary focus of these trials will be to validate the safety and tolerability of the drug.

MPS IIIA, also known as Sanfilippo Syndrome Type A, is a severe genetic disorder characterized by the accumulation of Heparan sulfate, leading to progressive damage to the central nervous system. The absence of Heparan N Sulfatase in patients triggers this accumulation, which, if untreated, can become life-threatening by the age of 15. The development of 'GC1130A' aims to address this deficiency through intracerebral ventricular enzyme replacement therapy (ICV ERT).

The collaboration between GC Biopharma and Novel Pharma has been focused on creating this innovative treatment, which has already shown a robust safety and efficacy profile in pre-clinical trials. This strong foundation helped 'GC1130A' secure Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from both the FDA and the European Medicines Agency (EMA).

The significance of this IND approval is profound. If successful, 'GC1130A' could become the first and only available treatment specifically for MPS IIIA, offering new hope to patients suffering from this debilitating condition. GC Biopharma's representative highlighted the importance of this development, stating that the company is dedicated to advancing to phase 1 clinical trials and is committed to providing new treatment options for patients.

GC Biopharma Corp., formerly known as Green Cross Corporation, is a leading biopharmaceutical company headquartered in Yongin, South Korea. The company has a rich history spanning over half a century and is renowned for its production of life-saving and life-sustaining protein therapeutics and vaccines. As one of the global leaders in plasma protein and vaccine products, GC Biopharma remains committed to delivering high-quality healthcare solutions.

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