GC Biopharma’s ERT GC-1130A designated orphan drug in EU for MPS IIIA
Jan. 23, 2024
The EMA has awarded orphan drug designation to GC Biopharma Corp.’s intracerebroventricular enzyme replacement therapy (ERT) candidate, GC-1130A, for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A), developed in collaboration with Novel Pharma Inc.