GC Biopharma (006280. KS) and Novel Pharma have announced that the U.S. FDA has granted Fast Track Designation for their jointly developed MPSIIIA (Sanfilippo syndrome Type A) treatment, GC1130A. This designation follows the FDA's clearance of the investigational new drug (IND) application for GC1130A last month.

With Fast Track Designation, the development process for GC1130A is set to be expedited. About Sanfilippo Syndrome Type A Sanfilippo syndrome Type A is a rare genetic disorder that causes central nervous system damage through the accumulation of heparan sulfate, leading to progressive neurological decline. Without treatment, patients face life-threatening complications by the age of 15.

GC1130A represents a potential breakthrough as the first and only treatment option specifically targeting MPSIIIA. GC1130A GC1130A is a biological drug that is being developed using high-concentration protein formulation technology, designed for administration to the central nervous system. It is delivered directly into the brain's ventricles through intracerebroventricular (ICV) injection, a method first applied globally by GC Biopharma's Hunter syndrome treatment, 'Hunterase', which has received market approval in Japan.

The potential of GC1130A to meet the unmet medical needs of Sanfilippo syndrome has been recognized by major drug regulatory agencies; in 2023, the FDA granted GC1130A Rare Pediatric Disease Designation (RPDD) and Orphan Drug Disease (ODD), and earlier this year, the European Medicines Agency (EMA) also granted GC1130A ODD status. Currently, GC Biopharma and Novel Pharma are preparing to initiate a multinational first-in-human clinical trial to evaluate the safety and tolerability of GC1130A in Korea, the US, and Japan.