FDA Approves Ultragenyx Gene Therapy FAYUVI for Sanfilippo Syndrome Type A

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โดย GlobeNewswire·US·Read original
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The U.S. Food and Drug Administration has approved FAYUVI, also known as UX111, a gene therapy developed by Ultragenyx Pharmaceutical Inc. for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare and progressive neurodegenerative lysosomal storage disorder. Abeona Therapeutics Inc. congratulated Ultragenyx on the approval, noting that the therapy originated from pioneering research by Drs. Haiyan Fu and Douglas McCarty at The Ohio State University and Nationwide Children's Hospital and was advanced through clinical development by Abeona as ABO-102 before Abeona out-licensed global development and commercialization rights to Ultragenyx in May 2022. Abeona Chief Executive Officer Vish Seshadri called the approval an extraordinary milestone for patients and families affected by a devastating disease that has lacked effective therapeutic options. The approval also represents a meaningful value-creation event for Abeona, which under its licensing agreement with Ultragenyx is eligible to receive certain commercial milestone payments and royalties tied to future product sales. FAYUVI is designed to deliver a functional copy of the SGSH gene to the central nervous system via an AAV9 viral vector, addressing the root genetic cause of MPS IIIA.

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