Andelyn Biosciences is manufacturing Ultragenyx’s newly FDA-approved Fayuvi gene therapy for commercial supply, marking a new commercial manufacturing milestone for the company.

FDA approves first MPS 3A gene therapy

The US Food and Drug Administration approved Fayuvi (rebisufligene etisparvovec-hopf) on September 17 for the treatment of neurologic manifestations of mucopolysaccharidosis type 3A (MPS 3A), also known as Sanfilippo syndrome type A, in paediatric patients with preserved neurodevelopmental function.

The approval makes Fayuvi the first FDA-approved treatment for Sanfilippo syndrome type A, a rare inherited disease that progressively damages the brain and nervous system. The FDA said children with the condition can lose cognitive, language and other developmental abilities over time.

Fayuvi is an adeno-associated virus (AAV) vector-based gene therapy administered as a single intravenous infusion.

Andelyn said it is manufacturing Fayuvi for commercial supply at its Columbus, Ohio facility. The company previously supported development and manufacturing of the programme and said the therapy is the first FDA-approved gene therapy manufactured using its AAV Curator Platform process.

The announcement follows Ultragenyx’s FDA approval and comes as the company prepares to move Fayuvi into commercial availability. Ultragenyx said commercial product is expected to be available to qualified treatment centres within 30 to 60 days.

Commercial manufacturing milestone

For Andelyn, the Fayuvi approval provides a commercial manufacturing milestone for its AAV gene therapy operations.

“We are proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator Platform process,” said Wade Macedone, chief executive officer of Andelyn Biosciences.

Macedone added that the milestone reflects the company’s focus on supporting gene therapy programmes through development and into commercial manufacturing.

Andelyn’s Columbus operations provide development and manufacturing services for cell and gene therapies, including process and analytical development and clinical and commercial manufacturing.

The company said its Curator Platform combines process development, manufacturing strategies and analytical capabilities for AAV programmes. However, the commercial approval of Fayuvi, rather than the platform itself, is the central development in the announcement.

Fayuvi brings first approved treatment for MPS IIIA

Fayuvi targets the neurological manifestations of MPS 3A, a rare lysosomal storage disease caused by a deficiency of the enzyme heparan N-sulfatase. The resulting accumulation of heparan sulfate contributes to progressive damage to the central nervous system.

The FDA said that, before Fayuvi’s approval, treatment for MPS 3A was limited to managing symptoms, with no FDA-approved therapy designed to alter the underlying course of the disease.

The approval therefore represents the more significant development behind Andelyn’s manufacturing announcement, while also highlighting the role of specialist CDMOs in moving AAV gene therapies from clinical development into commercial supply.

Fayuvi is now listed by the FDA among approved cellular and gene therapy products.