GC Biopharma Launches Japanese Clinical Trials For Sanfilippo Syndrome Treatment

GC Biopharma announced on Monday that it has received approval from Japan's Pharmaceuticals and Medical Devices Agency (PMDA) to initiate a phase 1 clinical trial for its investigational treatment, "GC1130A." This therapy, aimed at treating Mucopolysaccharidosis III type A (MPS-III-A), commonly known as Sanfilippo syndrome type A, is being co-developed with Novo Nordisk.

Sanfilippo syndrome is a rare and severe genetic disorder caused by a defective gene that leads to the accumulation of heparan sulfate in the body, resulting in progressive and debilitating damage, particularly to the brain. Most patients with this condition do not survive beyond their teenage years, and no approved treatments currently exist, highlighting a critical unmet need.

GC1130A is a pioneering therapy designed to combat Sanfilippo syndrome type A. The drug leverages GC Biopharma's advanced protein formulation technology to target the central nervous system effectively. It is administered directly into the brain's ventricles through intracerebroventricular injection, a method previously utilized in GC Biopharma's Hunter syndrome treatment, Hunterase, which is approved in Japan.

After securing a phase 1 investigational new drug (IND) approval from the U.S. Food and Drug Administration (FDA) in May and receiving a similar approval in Korea last month, GC Biopharma anticipates that the recent approval from Japan's PMDA will expedite the global clinical trial process for GC1130A.

A GC Biopharma official said, "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).”

GC1130A has been acknowledged by key international drug regulatory bodies as a groundbreaking therapy for the unmet medical need in treating Sanfilippo syndrome type A. In 2023, it was granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the U.S. FDA. Additionally, this year, the European Medicines Agency (EMA) awarded it an ODD. In June, the FDA further recognized the drug's potential by granting it Fast Track Designation.