BL 125845
BL 125845
U.S. License Holder:
Ultragenyx Pharmaceutical Inc.
Date of License:
September-17-2026
Last Update:
September-23-2026
FDA-Approved IndicationsFAYUVI (rebisufligene etisparvovec-hopf) is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function.
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