GC Cell

YONGIN, South Korea, May 21, 2024 /PRNewswire/ -- GC Biopharma (006280. KS) and Novel Pharma have announced that the U.S. FDA has cleared the investigational new drug (IND) application for their jointly developed MPSIIIA (Sanfilippo Syndrome Type A) treatment, GC1130A.

With this FDA clearance, the development of GC1130A is set to accelerate, with multinational clinical trials expected to commence this year. Clinical trial sites have been established in the United States, South Korea, and Japan, focusing on demonstrating the drug's safety and tolerability.

GC Biopharma and Novel Pharma have been co-developing this intracerebroventricular enzyme replacement therapy (ICVa ERT) for treatment for MPSIIIA patients who lack Heparan N Sulfatase.

GC1130A has received Orphan Drug Designation (ODD) from both FDA and EMA based on the robust safety and efficacy profile observed during preclinical studies. It also received Rare Pediatric Disease Designation (RPDD) from the FDA.

Sanfilippo Syndrome Type A is a genetic disorder that causes central nervous system damage through the accumulation of heparan sulfate, leading to progressive neurological decline. Without treatment, patients often face life-threatening complications by the age of 15. GC1130A represents a potential breakthrough as the first and only treatment option specifically targeting MPSIIIA.

GC Biopharma's representative further emphasized, "The IND clearance for GC1130A marks a significant milestone in our mission to bring hope to patients and families affected by Sanfilippo Syndrome. We are committed to advancing to phase 1 clinical trials and are dedicated to the development of new therapies, that address unmet medical needs.