Ultragenyx secures UK review for Sanfilippo gene therapy
Key points
- Ultragenyx Pharmaceutical (NASDAQ:RARE) received acceptance from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its UX111 gene therapy marketing authorisation application.
- The application covers mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A, a rare inherited disorder.
- Ultragenyx is coordinating the NICE assessment with the regulatory review, while the European Medicines Agency has also begun evaluating the application.
Ultragenyx Pharmaceutical (NASDAQ:RARE) secured acceptance of its UK marketing authorisation application for investigational gene therapy UX111 on October 28, 2026, advancing its regulatory review for Sanfilippo syndrome Type A.
The application covers mucopolysaccharidosis Type IIIA (MPS IIIA), a rare inherited metabolic disorder, and acceptance means the MHRA considers the submission sufficiently complete to begin formal scientific assessment, not that the treatment has been approved.
The company has aligned its NICE evaluation with the marketing application review, while NICE previously directed UX111 to its Highly Specialised Technologies programme after determining that the treatment met all four eligibility criteria.
The European Medicines Agency also began its application review in October 2026.
Ultragenyx aims to coordinate the regulatory and health technology assessment processes so that a potential NICE funding appraisal decision can follow as closely as possible after a possible UK marketing authorisation, although both decisions remain subject to their respective reviews.
Ultragenyx develops medicines for rare and serious genetic diseases, including metabolic disorders, with its research programmes covering gene therapies and other treatments for conditions with limited treatment options.
Its UX111 programme focuses on delivering a functional copy of the SGSH gene to address the underlying genetic deficiency associated with MPS IIIA, while the company pursues regulatory reviews in the United Kingdom and European Union.
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