JCR Bags FDA’s Orphan Tag for MPS IIIA Drug in US

December 15, 2023
JCR Pharmaceuticals said on December 14 that the US FDA has granted orphan drug designation to JR-441, an enzyme therapy for mucopolysaccharidosis type III A (MPS IIIA), also known as Sanfilippo syndrome type A. The FDA designation comes after the...read more