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Ultragenyx announced the discontinuation of its GTX-102 Angelman syndrome program and its UX701 Wilson disease program following unsuccessful clinical trial results.
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The UK Medicines and Healthcare products Regulatory Agency (MHRA) has accepted the Marketing Authorisation Application for Ultragenyx's investigational gene therapy, rebisufligene etisparvovec, for the treatment of MPS IIIA.
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.
Ultragenyx cuts 20% of staff and trims pipeline programs after missing Q3 targets, aiming for 2027 profitability through new commercial launches.
Ultragenyx Pharmaceutical Inc. reported its Q3 2026 financial results, highlighting a narrowed net loss, revenue growth, and a planned 20% workforce reduction to support its path to profitability in 2027.