GC Biopharma said Monday that it has received approval from the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) for a phase 1 clinical trial notification (CTN) for “GC1130A,” a treatment for Mucopolysaccharidosis III type A (MPS-III-A), also known as Sanfilippo syndrome type A, being developed in collaboration with Novo Nordisk.

Sanfilippo syndrome (MPS) is a recessive genetic disorder caused by a gene defect that leads to a buildup of heparan sulfate in the body, which causes progressive damage. It is a severe and rare disease characterized by severe brain damage, with most patients dying around the age of 15. There are no licensed therapies for the disease, representing a significant unmet medical need for patients.

GC1130A is a first-in-class drug being developed to treat Sanfilippo syndrome (type A) by applying GC Biopharma’s highly concentrated protein formulation technology to the central nervous system. This technology enables the drug to be administered directly into the brain's ventricles for improved therapeutic effectiveness (Intracerebroventricular injection). This method has been applied to GC Biopharma’s Hunterase, a Hunter syndrome treatment approved in Japan.

Following GC1130A's phase 1 investigational new drug (IND) approval from the U.S. Food and Drug Administration (FDA) in May and its Korean IND approval last month, GC Biopharma expects the Japanese PMDA approval to accelerate the multinational clinical development of GC1130A.

"As this is a disease with a large unmet medical need, we will do our best to develop new drugs to bring hope to patients suffering from Sanfilippo syndrome (type A)," a GC Biopharma official said.

GC1130A has won recognition from major overseas drug regulatory agencies as an innovative new drug to address the unmet medical need for Sanfilippo syndrome (type A). It received an Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. FDA in 2023 and an ODD from the European Medicines Agency (EMA) this year. In June, it also received Fast Track Designation from the FDA.