Overview
- The FDA approval on Thursday, Sept. 17, 2026, covers pediatric patients with Sanfilippo syndrome Type A and rests on up to eight years of clinical data showing treated children maintained or improved cognitive function versus untreated patients.
- Fayuvi is given as a single intravenous infusion that uses an AAV9 viral vector to deliver a working SGSH gene so the body can make sulfamidase and clear toxic heparan sulfate from the brain.
- Ultragenyx set a U.S. list price of $3.95 million and said the therapy will be available only at trained Qualified Treatment Centers, with initial shipments to those centers expected in 30 to 60 days.
- The company manufactures Fayuvi at sites in Massachusetts and Ohio, a limited-capacity model that, together with restricted center-based delivery, shapes access and the small commercial footprint analysts expect.
- Investors reacted positively, lifting Ultragenyx shares about 12–13%, and analysts project peak worldwide sales in the low hundreds of millions while families may see lasting clinical benefit that could reduce long-term care needs.