poisar

Stocks · News reference

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.

More stocks newsConnected markets

Reference Details

Connected Markets

Poisar stores the source's headline, summary and market tags for reference. Reporting and full article text remain with the original publisher.

RARE
Source
fiscal.ai
Observed
Checked
Cadence
Captured once, then retained as an archive reference
Status
fresh

Keep reading

  1. Na

    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.

    RARE