CLEVELAND, Ohio — September 18, 2026
Abeona Therapeutics congratulated Ultragenyx Pharmaceutical on the U.S. FDA approval of FAYUVI™ (rebisufligene etisparvovec-hopf), formerly UX111, for the treatment of patients with Sanfilippo syndrome type A, also known as mucopolysaccharidosis IIIA (MPS IIIA). The approval represents a major regulatory milestone for the rare pediatric neurodegenerative disease and follows years of research and development involving Abeona and its academic collaborators. The program originated from research conducted at The Ohio State University and Nationwide Children’s Hospital before being advanced by Abeona as ABO-102.
FAYUVI Program Advances From Academic Research to Approval
Abeona advanced the program through key preclinical and clinical development stages before entering an out-licensing agreement with Ultragenyx in 2022. Under the agreement, Ultragenyx received global development and commercialization rights to the therapy and subsequently advanced the program through late-stage development and regulatory review. Abeona said its work helped establish foundational clinical data supporting continued development, while Ultragenyx provided the resources and capabilities required to progress the therapy toward regulatory approval and commercialization.
Gene Therapy Targets Root Cause of MPS IIIA
FAYUVI is designed to address the underlying genetic cause of MPS IIIA by delivering a functional copy of the SGSH gene to the central nervous system using an AAV9 viral vector. MPS IIIA is caused by deficiency of the N-sulfoglucosamine sulfohydrolase (SGSH) enzyme, resulting in accumulation of heparan sulfate and progressive damage, particularly in the central nervous system. Children affected by the disease can experience developmental regression, loss of motor skills and premature death, highlighting the substantial unmet medical need associated with the condition.
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FDA Approval Creates Commercial Milestone for Abeona
The FAYUVI approval also represents a potential value-creation milestone for Abeona, which is eligible for certain commercial milestone payments and royalties on future product sales under its licensing agreement with Ultragenyx. Abeona continues to operate as a commercial-stage cell and gene therapy company, with its approved ZEVASKYN® (prademagene zamikeracel) for wounds associated with recessive dystrophic epidermolysis bullosa and a development portfolio that includes ABO-701, an engineered T-cell therapy targeting PSMA. The FAYUVI milestone highlights the potential long-term commercial impact of programs originating from Abeona’s earlier research and development activities.
Source : Abeona Therapeutics, press release
