- Andelyn Biosciences has begun manufacturing Fayuvi™ (rebisufligene etisparvovec-hopf) for commercial supply at its Columbus, Ohio facility following FDA approval for the treatment of Sanfilippo syndrome type A.
- Fayuvi is the first FDA-approved gene therapy manufactured using Andelyn’s AAV Curator® Platform process.
Andelyn Biosciences, a cell and gene therapy contract development and manufacturing organization (CDMO), has begun manufacturing Fayuvi™ (rebisufligene etisparvovec-hopf) for commercial supply at its facility in Columbus, Ohio. The announcement follows the U.S. Food and Drug Administration’s approval of Fayuvi for the treatment of Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA).
Fayuvi is the first FDA-approved gene therapy manufactured using Andelyn’s AAV Curator® Platform process. The company said its commercial manufacturing activities reflect the process development, scientific and quality systems, and cross-functional capabilities required to manufacture the gene therapy for commercial use.
Sanfilippo syndrome type A is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is characterized by rapid neurodegeneration beginning in early childhood. The disease is estimated to affect 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years.
Andelyn’s AAV Curator® Platform uses a configurable, data-informed approach to AAV process development and manufacturing. The platform integrates process knowledge, modular manufacturing strategies and fit-for-purpose analytics to support gene therapy programs from development through commercial launch.
“We are proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator® Platform process. This milestone reflects the purpose behind Andelyn’s founding and underscores the importance of building commercial readiness through rigorous science, accountable execution, and quality at every stage.”
Wade Macedone, Chief Executive Officer of Andelyn Biosciences
