CDMO Andelyn Biosciences is now manufacturing Fayuvi™ (rebisufligene etisparvovec-hopf), an FDA-approved gene therapy for commercial supply at its Columbus, OH facility. The FDA last week approved the product developed by Ultragenyx Pharmaceutical for the treatment of Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA (MPS IIIA). Fayuvi is also being manufactured ay Ultragenyx’s gene therapy manufacturing facility in Bedford, MA.
Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease that primarily affects the central nervous system (CNS) and is marked by rapid neurodegeneration beginning in early childhood. MPS IIIA is estimated to affect 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years.
Fayuvi is the first FDA-approved gene therapy manufactured using the Andelyn AAV Curator® Platform process.
“We congratulate Ultragenyx on this significant achievement and recognize the MPS IIIA community, investigators, and everyone who contributed to advancing this program,” said Wade Macedone, CEO of Andelyn Biosciences. “We are proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator Platform process.”
Andelyn’s AAV Curator Platform uses a configurable, data-informed approach to AAV process development and manufacturing, according to Macedone, who added that it integrates process knowledge, modular manufacturing strategies, and fit-for-purpose analytics to support gene therapy programs from development through commercial launch.
