FDA Grants Fast Track Status to GC Biopharma's Sanfilippo Syndrome Drug
GC Biopharma is currently preparing clinical trials to evaluate the safety and tolerability of Sanfilippo Syndrome Type A treatment, “GC1130A” in South Korea, the United States, and Japan.
GC Biopharma announced on June 10 that their Sanfilippo Syndrome Type A treatment, “GC1130A,” co-developed with Nobel Pharma, has received Fast Track Designation from the U.S. Food and Drug Administration (FDA). This designation is a significant milestone in the development of this promising new therapy for a rare and devastating genetic disorder.
Sanfilippo Syndrome Type A, also known as MPS IIIA, is a rare genetic disorder caused by a defect in a specific gene, leading to the accumulation of heparan sulfate in the body. This accumulation causes progressive damage, particularly to the brain, and most patients die around the age of 15 due to severe brain damage. Currently, there are no approved treatments for this condition, making the development of GC1130A particularly crucial.
GC1130A is a biopharmaceutical drug developed using GC Biopharma's high-concentration protein formulation technology. It is designed to be administered directly into the brain's ventricles through an intracerebroventricular injection (ICV) to enhance therapeutic effects. This method was first applied to their Hunter Syndrome treatment, “Hunterase,” which received marketing approval in Japan.
Last month, the FDA approved the Investigational New Drug (IND) application for GC1130A, allowing the commencement of Phase 1 clinical trials. This approval was followed by the Fast Track Designation, which is intended to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need.
In 2023, GC1130A was also designated as an Orphan Drug (ODD) and a Rare Pediatric Disease Drug (RPDD) by the U.S. FDA. Additionally, it received Orphan Drug Designation from the European Medicines Agency (EMA) this year. These designations provide certain benefits, such as market exclusivity and financial incentives, to encourage the development of treatments for rare diseases.
June 13, 2024
