Abeona gains milestone from FAYUVI approval
- Abeona Therapeutics (NASDAQ:ABEO) said Ultragenyx received FDA approval for FAYUVI, a gene therapy for Sanfilippo syndrome type A.
- FAYUVI originated from Abeona’s ABO-102 program before being licensed to Ultragenyx in 2022.
- Abeona is eligible for commercial milestone payments and royalties tied to future FAYUVI sales.
Abeona Therapeutics (NASDAQ:ABEO) announced that Ultragenyx Pharmaceutical received U.S. Food and Drug Administration approval for FAYUVI (rebisufligene etisparvovec-hopf), a gene therapy for Sanfilippo syndrome type A.
The therapy, also known as UX111, originated from Abeona’s ABO-102 program, which was developed from research conducted by Drs. Haiyan Fu and Douglas McCarty at The Ohio State University and Nationwide Children’s Hospital.
In 2022, Abeona licensed global development and commercialization rights for the program to Ultragenyx, and the FDA approval triggers eligibility for Abeona to receive commercial milestone payments and royalties tied to future FAYUVI product sales.
Abeona develops gene and cell therapies for rare diseases, while the FAYUVI program targets Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA), a rare progressive neurodegenerative lysosomal storage disorder.
The approval represents a regulatory milestone for the program and establishes a commercial pathway for FAYUVI under Ultragenyx’s development and commercialization rights.
