Ultragenyx Gene Therapy for MPS IIIA Advances to EMA Review

Ultragenyx Pharmaceutical Inc. announced that the European Medicines Agency (EMA) has validated its Marketing Authorisation Application (MAA) for rebisufligene etisparvovec. This investigational gene therapy targets mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A. The validation confirms the application's completeness, allowing formal scientific review to begin for this potential new treatment.