Wedbush Maintains Neutral on Ultragenyx Pharmaceutical, Lowers Price Target to $22

Ultragenyx Announces UK MHRA Acceptance of Marketing Authorisation Application for Rebisufligene Etisparvovec

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 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)

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.

Barclays Maintains Overweight on Ultragenyx Pharmaceutical, Lowers Price Target to $32

Ultragenyx Discontinues GTX-102 and UX701 Programs Following Clinical Trial Outcomes

Ultragenyx announced the discontinuation of its GTX-102 Angelman syndrome program and its UX701 Wilson disease program following unsuccessful clinical trial results.

Ultragenyx Pushes Toward Profitability in 2027, Led by Pruned Pipeline, Layoffs

Ultragenyx cuts 20% of staff and trims pipeline programs after missing Q3 targets, aiming for 2027 profitability through new commercial launches.

Ultragenyx Reports Third Quarter 2026 Financial Results and Announces Expense Reductions

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.