Business, energy, technology, markets and global industry news from Business News Today

FAYUVI

PharmaDeviceNews FAYUVI
Ultragenyx Pharmaceutical’s $3.95 million FAYUVI gene therapy is the first FDA-approved disease-modifying treatment for children with Sanfilippo syndrome type A. Representative image.
Pharma & Biotech

Why Ultragenyx stock jumped after FDA approved $3.95 million Fayuvi gene therapy

Ultragenyx Pharmaceutical has secured full FDA approval for Fayuvi, the first treatment for Sanfilippo syndrome Type A, giving the rare-disease biotech a major commercial catalyst only weeks after a Phase 3 setback erased nearly half its market value.

Read More
Ultragenyx Pharmaceutical’s $3.95 million FAYUVI gene therapy is the first FDA-approved disease-modifying treatment for children with Sanfilippo syndrome type A. Representative image.
Features & Analysis

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

Read More