GC Biopharma Corp. (006280.KS), a South Korea-based biopharmaceutical company, announced on Monday that it has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate, GC1130A, engineered for Sanfilippo Syndrome type A (mucopolysaccharidosis type IIIA), and produced in partnership with Novel Pharma.

GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the US 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 sulphate, leading to a progressive neurodegeneration in paediatric 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 sulphate and restoring brain function.

GC1130A has previously achieved notable milestones by securing both Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the US FDA in January 2023.