Trial Profile

Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA

Status: Recruiting

Phase of Trial: Phase II/III

Latest Information Update: 27 Sep 2026

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At a glance

17 Sep 2026 According to an Ultragenyx Pharmaceutical media release,company will host a conference call today at 5:30 p.m. Eastern Time/2:30 p.m. Pacific Time to discuss the FAYUVI approval.
17 Sep 2026 According to a U.S. Food and Drug Administration (FDA) media release, FDA today approved Fayuvi (rebisufligene etisparvovec-hopf), the first treatment for pediatric patients with mucopolysaccharidosis type IIIA. The approval underscores the Office of Therapeutic Products (OTP) and FDA's commitment to applying rigorous evidentiary standards as the field of gene therapy continues to advance.
11 Jun 2026 Planned End Date changed from 1 Jul 2027 to 1 Mar 2029.

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