Ultragenyx gains EMA validation for Sanfilippo gene therapy

• Ultragenyx (NASDAQ:RARE) said the European Medicines Agency validated its marketing authorisation application for rebisufligene etisparvovec.
• Validation means the filing is complete enough for formal scientific review, but it does not represent approval or a finding on safety or efficacy.
• Ultragenyx is also pursuing broader geographic expansion, including regulatory engagement in the UK and Saudi Arabia.

Ultragenyx (NASDAQ:RARE) announced that the European Medicines Agency validated its marketing authorisation application for investigational gene therapy rebisufligene etisparvovec to treat Sanfilippo syndrome Type A.

The validation confirms that the application contains sufficient information to begin the EMA’s formal scientific review process, but it does not constitute regulatory approval.

Rebisufligene etisparvovec has received PRIME and Orphan Drug designations in Europe, which are intended to support development and regulatory evaluation without guaranteeing authorisation.

The application will also be assessed under the European Union’s Joint Clinical Assessment framework, which coordinates clinical evaluation across member states.

Ultragenyx said it plans to expand the therapy’s regulatory reach beyond the European Union and has started engagement with the UK’s Medicines and Healthcare products Regulatory Agency.

The company has also begun regulatory discussions with Saudi Arabia’s Food and Drug Authority as it pursues potential access to additional international markets.