Andelyn reaches commercial manufacturing milestone following FDA approval of Fayuvi

Fayuvi (rebisufligene etisparvovec-hopf) was approved by the FDA on September 17 for the treatment of neurologic manifestations of mucopolysaccharidosis type IIIA (MPS IIIA), also known as Sanfilippo syndrome type A, in pediatric patients with preserved neurodevelopmental function. It is the first FDA-approved treatment for the rare inherited disorder.

Andelyn is manufacturing Fayuvi for commercial supply at its facility in Columbus, Ohio. Ultragenyx has confirmed that the therapy is manufactured in the US at both Andelyn’s Columbus facility and Ultragenyx’s Gene Therapy Manufacturing Facility in Bedford, Massachusetts.

The approval also marks the first FDA-approved gene therapy manufactured using Andelyn’s AAV Curator® Platform, according to the company.

Gene therapy for Sanfilippo syndrome type A

Sanfilippo syndrome type A is a rare, inherited lysosomal storage disease that primarily affects the central nervous system. The condition is associated with progressive neurodegeneration beginning in early childhood, with children typically experiencing developmental delays followed by loss of cognitive, language and motor function.

The disease is caused by a deficiency of the sulfamidase enzyme, resulting in the accumulation of heparan sulfate and progressive damage to the central nervous system.

Ultragenyx estimates that Sanfilippo syndrome type A affects approximately 3,000 to 5,000 patients in commercially accessible geographies, with a median life expectancy of 15 years.

Fayuvi is a single-dose intravenous AAV9 gene therapy designed to deliver a functional copy of the gene responsible for producing the deficient SGSH enzyme. The FDA said the therapy provides a working copy of the SGSH gene, enabling cells to produce sulfamidase and break down heparan sulfate.

First approved therapy using Andelyn's AAV Curator Platform

For Andelyn, the approval represents the first FDA-approved gene therapy manufactured using its AAV Curator Platform.

The company describes the platform as a configurable, data-informed approach to AAV process development and manufacturing. It combines process knowledge, modular manufacturing strategies and fit-for-purpose analytics to support gene therapy programmes from development through commercial launch.

“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,” said Wade Macedone, CEO of Andelyn Biosciences.

Andelyn said its commercial manufacturing of Fayuvi reflects the process development, quality systems and cross-functional work required to bring a gene therapy programme through to commercial supply.

A milestone for AAV manufacturing

The Fayuvi approval also highlights the manufacturing infrastructure supporting the growing gene therapy sector, as programmes move from clinical development towards approved products and commercial supply.

Ultragenyx said Fayuvi is expected to be available for shipment to qualified treatment centres within 30–60 days of approval. The therapy will be distributed through a network of US-based Qualified Treatment Centers with specialised expertise in administering gene therapies.

For Andelyn, the approval provides a first commercial example of its AAV Curator Platform being used in the manufacture of an FDA-approved gene therapy.

The company said it will continue to support other late-stage gene therapy programmes through its development and commercial manufacturing capabilities.

Fayuvi's approval marks a step forward for patients with Sanfilippo syndrome type A, while also providing a notable commercial manufacturing milestone for Andelyn and its AAV Curator Platform.