Ultragenyx secures first gene therapy approval for GSDIa as Sanfilippo asset decision pending

Ultragenyx received accelerated FDA approval for its gene therapy DTX401, marketed as Genglycos, to treat glycogen storage disease type Ia (GSDIa).

The approval comes as the company awaits a second FDA decision on its Sanfilippo gene therapy UX111, which was rejected last summer, with a ruling expected around Sept 19.

Analysts said the GSDIa clearance de-risks the pending Sanfilippo asset and highlights Ultragenyx's ability to secure back-to-back authorizations. DTX401 uses an adeno-associated virus vector to deliver the missing gene and aims to lower daily cornstarch intake for patients eight years and older.