Ultragenyx receives FDA approval for Sanfilippo syndrome gene therapy

Ultragenyx Pharmaceuticals has received FDA approval for Fayuvi (rebisufligene etisparvovec-hopf), the first approved treatment for Sanfilippo syndrome Type A. This rare, fatal genetic disorder causes progressive neurodegeneration by preventing the body from clearing toxic sugar molecules in the brain.

Fayuvi is a one-time gene therapy administered via intravenous infusion. Clinical trials indicated that treated children showed maintained or improved cognitive function compared to untreated peers. Ultragenyx has set a US list price of $3.95 million for the therapy.

Abeona Therapeutics, which originally developed the therapy as ABO-102 before licensing it to Ultragenyx in 2022, is set to receive mid-single-digit royalties and up to $30 million in commercial milestone payments. Ultragenyx expects the product to reach treatment centers within 30 to 60 days.