{"url":"https://newsforagent.com/news/2026-11-06/ultragenyx-mhra-review-mps-iiia-gene-therapy","markdown_url":"https://newsforagent.com/news/2026-11-06/ultragenyx-mhra-review-mps-iiia-gene-therapy.md","title":"Ultragenyx UK Gene Therapy Application Enters MHRA Review","headline":"Ultragenyx's MPS IIIA Gene Therapy Application Accepted for Review by UK MHRA","description":"Ultragenyx's marketing application for its MPS IIIA gene therapy is now under MHRA review, with a NICE evaluation timed to a potential UK approval.","section":"Regulation & Approvals","published_at":"2026-11-06T08:56:38+00:00","modified_at":"2026-11-06T08:56:38+00:00","author":"News Desk","key_takeaways":["The UK MHRA accepted Ultragenyx's Marketing Authorisation Application for rebisufligene etisparvovec on October 28, 2026.","Ultragenyx agreed to schedule the NICE evaluation alongside the MHRA review, aiming for a decision close to potential approval.","The FDA approved rebisufligene etisparvovec on September 17, 2026, while the therapy remains investigational in the UK."],"companies":[{"name":"Ultragenyx Pharmaceutical","is_primary":true,"profile_url":"https://akta.pro/company-profiles/0000dvt/ultragenyx-pharmaceutical","website":"http://www.ultragenyx.com"},{"name":"National Institute for Health and Care Excellence (NICE)","is_primary":false,"profile_url":"https://akta.pro/company-profiles/00067bl/national-institute-for-health-and-care-excellence-nice","website":"http://www.nice.org.uk"}],"source":{"publisher":"TradingView","title":"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)","url":"https://www.tradingview.com/news/globenewswire:9434a0bd5094b:0-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/"},"other_sources":[],"tags":["Regulation & Approvals","Ultragenyx Pharmaceutical","National Institute for Health and Care Excellence (NICE)"],"body_markdown":"Ultragenyx Pharmaceutical's Marketing Authorisation Application for its investigational gene therapy rebisufligene etisparvovec, also known as UX111, is under review by the UK Medicines and Healthcare products Regulatory Agency, the company said in a [TradingView](https://www.tradingview.com/news/globenewswire:9434a0bd5094b:0-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/) release. The MHRA accepted the application on October 28, 2026.\n\n## Ultragenyx's MHRA Filing for Sanfilippo Type A\n\n[Ultragenyx Pharmaceutical](https://akta.pro/company-profiles/0000dvt/ultragenyx-pharmaceutical) is a biopharmaceutical company developing and commercialising therapies for rare and ultra-rare genetic diseases. The filing covers rebisufligene etisparvovec, a single-dose intravenous AAV9 gene therapy designed to deliver a functional copy of the gene for the deficient SGSH enzyme.\n\nThe MHRA's acceptance confirms the application is complete enough for formal scientific review of quality, safety and efficacy. The therapy remains investigational in the UK, and the company said there can be no assurance that marketing authorisation will be granted.\n\n\"The acceptance of our application by the MHRA 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. She said the company agreed to schedule the NICE evaluation to align with the MHRA review, with the ambition of a NICE decision as close as possible to a potential regulatory approval.\n\nNICE appraises clinical and cost-effectiveness to inform NHS funding decisions in England. Its Prioritisation Board previously routed the treatment to the Highly Specialised Technology programme after agreeing it meets all four criteria of that framework.\n\n## Ultragenyx's Earlier FDA and EMA Steps\n\nAccording to akta.pro [company records](https://akta.pro/company-data), the FDA accepted the BLA resubmission for UX111 in April 2026 with a PDUFA action date of September 19, 2026. The company received FDA approval for rebisufligene etisparvovec on September 17, 2026, the release states.\n\nUltragenyx also announced last month the start of the European Medicines Agency Marketing Authorisation Application review. Stock Titan reported on October 2, 2026 that the EMA validated the MPS IIIA gene therapy application, enabling formal scientific review without constituting approval.\n\n## What Comes Next for the UK Filing\n\nThe company said it is working toward a NICE decision timed as closely as possible to a potential MHRA decision. Ultragenyx also said it is in discussions with the Saudi Food and Drug Authority as part of its regulatory strategy.\n","publisher":{"name":"News for Agent","url":"https://newsforagent.com"}}