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)
Key Highlights
- ➤Ultragenyx’s MHRA application for UX111 accepted October 28, 2026, beginning formal review
- ➤UX111 remains investigational in the UK; marketing authorisation is not guaranteed
- ➤EMA began reviewing Ultragenyx’s UX111 application last month
- ➤NICE evaluation scheduled alongside MHRA review to align potential NHS funding decision
Expert Statements
Jane Cooper, Senior Vice President and Region Head for Europe, Middle East and Africa at Ultragenyx
“The acceptance of our application by the MHRA marks an important regulatory milestone in the ongoing review process for this investigational therapy”
Jane Cooper, Senior Vice President and Region Head for Europe, Middle East and Africa at Ultragenyx
“We recognise the profound unmet need within the Sanfilippo syndrome Type A community and are working with urgency to advance our regulatory strategy.”
Jane Cooper, Senior Vice President and Region Head for Europe, Middle East and Africa at Ultragenyx
“To enable sustainable access on the NHS for eligible children, we have agreed to schedule the NICE evaluation to align with the Marketing Authorisation Application review with the ambition to receive a NICE decision as close as possible to a potential regulatory approval”
BASEL, Switzerland, Nov. 06, 2026 (GLOBE NEWSWIRE) -- Ultragenyx (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultra-rare genetic diseases, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) accepted on 28(th) October 2026 the Company’s Marketing Authorisation Application (MAA) for rebisufligene etisparvovec (also known as rebi-vec or UX111), an investigational gene therapy for the treatment of mucopolysaccharidosis Type IIIA (MPS IIIA), also known as Sanfilippo syndrome Type A.
Sanfilippo syndrome Type A is a rare, progressive, inherited disorder associated with severe neurological impairment and significant unmet medical need. Currently, treatment options remain limited for affected children and their families.
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