GC Biopharma said it has submitted an investigational new drug (IND) application and applied for Fast Track Designation with the U.S. FDA for GC1130A, an intracerebroventricular (ICV) enzyme replacement therapy (ERT) drug for Sanfilippo Syndrome Type A (MPS IIIA), jointly developed with Korean biotech venture Novel Pharma.

GC Biopharma has applied for IND and Fast Track Designation for its Sanfilippo Syndrome treatment candidate, GC1130A, to the U.S. Food and Drug Administration.
GC Biopharma has applied for IND and Fast Track Designation for its Sanfilippo Syndrome treatment candidate, GC1130A, to the U.S. Food and Drug Administration.

MPS IIIA is characterized by the accumulation of heparan sulfate due to a genetic defect, leading to progressive damage and severe brain impairment.

Most affected individuals succumb to the disease around the age of 15, and there are currently no approved treatments available, highlighting a substantial unmet need, according to GC Biopharma.

The FDA's Fast Track Designation is designed to expedite the development of drugs that address significant unmet medical needs in treating serious conditions. If granted, it facilitates more frequent communication with the FDA, potentially accelerating the drug's development and review process.

GC Biopharma and Novel Pharma are developing an ERT that involves the ICV of heparan N-sulfatase, an enzyme not naturally expressed in patients.

The approach has shown promise in preclinical stages, demonstrating efficacy and safety. It has received an Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the FDA and has also won an ODD from the European Medicines Agency (EMA).

"Through our collaboration with Novel Pharma, we are eager to accelerate the development of this new drug by entering clinical trials promptly, offering hope to patients worldwide," a GC Biopharma official said.

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