Ultragenyx Announces Marketing Authorisation Application (MAA) Submission to the European Medicines Agency (EMA) for the First Investigational Gene Therapy for MPS IIIA (Sanfilippo Syndrome Type A)

BASEL, Switzerland, Oct. 02, 2026 (GLOBE NEWSWIRE) -- Today Ultragenyx announced that the European Medicines Agency (EMA) has validated the Company's Marketing Authorisation Application (MAA) for its investigational gene therapy, rebisufligene etisparvovec, for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A.

EMA validation confirms that the application is sufficiently complete for the Agency to begin its formal scientific review. Validation does not constitute approval of the application, nor does it represent a determination regarding the safety or efficacy of the investigational therapy, nor guarantee that the application will ultimately receive marketing authorisation. "The validation of our application by the EMA marks an important regulatory milestone in the ongoing review process for this investigational therapy," said Jane Cooper, Senior Vice President and Region Head for Europe, Middle East and Africa at Ultragenyx. "We recognize the significant unmet need within the MPS IIIA community and continue to advance our global regulatory strategy with the European Medicines Agency (EMA). We also plan to expand to other geographies over time.”