FAYUVI costs $3.95m for one infusion. Can Ultragenyx make the first Sanfilippo therapy accessible?
Ultragenyx Pharmaceutical has won FDA approval for FAYUVI, the first treatment designed to alter the course of Sanfilippo syndrome type A. The one-time AAV9 gene therapy carries a $3.95 million U.S. list price and will initially flow through specialist treatment centres, putting early diagnosis, payer access and preservation of neurodevelopmental function at the centre of