FDA approves Fayuvi, the first treatment for Sanfilippo syndrome type A, at $3.95M per patient

Ultragenyx/Fayuvi (UX111)

The FDA fully approved Fayuvi (UX111), a one-time AAV9 gene therapy, as the first treatment for MPS IIIA, a fatal inherited brain disease. Children treated at ages 2-5 kept or improved cognitive scores, while untreated children plateau or decline. The wholesale price is $3.95M, and the approval brought the company's second priority review voucher of 2026.

• Standard full approval rather than accelerated approval.
• Wholesale price $3.95M; first shipments expected within 30-60 days.
• Made at Ultragenyx's Bedford, Massachusetts plant and by Andelyn Biosciences.
• Originally Abeona's ABO-102, licensed to Ultragenyx in 2022.
• Comes with a priority review voucher; recent vouchers have sold for about $200M.