Ultragenyx says EMA validates MAA for MPS IIIA gene therapy rebisufligene etisparvovec
RARE•The EMA validated Ultragenyx’s marketing authorisation application for investigational MPS IIIA gene therapy rebisufligene etisparvovec, starting a formal scientific review but not granting marketing authorisation.
1. Formal review begins
The EMA validated Ultragenyx’s application for rebisufligene etisparvovec, an investigational gene therapy for MPS IIIA. The validation begins the agency’s formal scientific review and is not a marketing authorisation decision. The therapy holds EMA PRIME status, which supports an accelerated regulatory pathway for the rare neurodegenerative disorder.
