FDA approves Ultragenyx gene therapy FAYUVI for Sanfilippo syndrome type A

The U.S. FDA granted standard full approval to Ultragenyx’s gene therapy FAYUVI (rebisufligene etisparvovec-hopf), also known as UX111, for pediatric patients with Sanfilippo syndrome type A (MPS IIIA).

It is described as the first FDA-approved treatment for Sanfilippo syndrome type A, a rare and progressive neurodegenerative disease.

The approval was supported by data from the Transpher A trial and long-term follow-up studies showing clinical benefit, durable effect, and an acceptable safety profile.

FAYUVI is an adeno-associated virus (AAV) gene therapy designed to deliver a functional copy of the SGSH gene.

Ultragenyx said the therapy should become available at specialized U.S. treatment centers within about 30 to 60 days, and the company set a U.S. list price of $3.95 million.

Sources:

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