GC Biopharma Corp. announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate, GC1130A, designed for Sanfilippo Syndrome type A (mucopolysaccharidosis type IIIA), developed in collaboration with Novel Pharma.

GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023. Building on these accomplishments, the latest approval in Europe adds another significant achievement.

Sanfilippo Syndrome (type A) is a genetic disorder that triggers central nervous system damage by accumulating Heparan sulfate, leading to a progressive neurodegeneration in pediatric population. Patients with MPS IIIA typically have a life expectancy of approximately 15 years, and as of now, there are no approved therapies available. In nonclinical studies, GC1130A demonstrated both safety and efficacy in eliminating Heparan sulfate and restoring brain function.

Since 2020, GC Biopharma and Novel Pharma have collaborated on the development of GC1130A. Utilizing proprietary recombinant protein manufacturing technology, GC Biopharma has successfully formulated GC1130A to be compatible with cerebrospinal fluid at high protein concentrations. Novel Pharma, a biotech company with expertise in rare disease drug development in Korea.

"This EMA designation further underlines the potentials of our collaborative pipeline in addressing the disease pathology in upcoming clinical trials", said GC Biopharma and added that it will be "committing to expeditiously advancing into clinical trials, acknowledging the urgent, unmet medical needs of Sanfilippo Syndrome patients."