Ultragenyx Jumps After FDA Approval for $2.7M Gene Therapy
Ultragenyx’s one-time gene therapy becomes the first approved treatment targeting the underlying cause of GSDIa. The approval follows trial results showing a 61% mean reduction from baseline at Week 96, and a 30 to 60 day path to qualified treatment centers. The U.S. list price is about $2.7 million per patient for an estimated 1,500 to 2,500 Americans.