Ultragenyx Announces the Marketing Authorisation Application (MAA) for its Investigational Gene Therapy for MPS IIIA (Sanfilippo syndrome Type A) is Under Review with the UK Medicines and Healthcare Products Regulatory Agency (MHRA)

Ultragenyx announced that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) accepted its Marketing Authorisation Application for rebisufligene etisparvovec (UX111), an investigational gene therapy for MPS IIIA (Sanfilippo syndrome Type A). The company has also coordinated with NICE to align the evaluation timeline with the regulatory review process. The therapy received FDA approval in September 2026 and is also under review by the European Medicines Agency.