The CDMO will commercially manufacture the lentiviral vector and genetically modified cell drug product (DP) of Lenmeldy, which became the first therapy to receive FDA approval for MLD on March 18 2024.
It is intended for the treatment of pre-symptomatic late infantile, pre-symptomatic early juvenile and early symptomatic early juvenile MLD.
“It is wonderful to see Orchard Therapeutics reach this goal. This accomplishment is a strong proof point of our commitment at AGC Biologics to collaborating directly with developers to meet rigorous regulatory standards and achieve the best possible results for our partners,” says Patricio Massera, CEO of AGC Biologics.
With this announcement, AGC Biologics’ Milan location becomes the only global CDMO site to receive the regulatory agency’s clearance to produce this treatment for patients in the U.S. commercially.
"AGC Biologics has played a pivotal role as a strategic partner throughout the clinical development and commercial scale-up of atidarsagene autotemcel, which helped facilitate regulatory approvals in Europe and the United States" said Nicoletta Loggia, chief technical officer of Orchard Therapeutics.
"Utilizing our best-in-class commercial HSC gene therapy manufacturing platform, we have consistently met the demands for drug product production since the European launch in early 2021.
“This has enabled us to provide treatment to patients from three continents, including Europe on a commercial basis, the Middle East through treatment abroad programs, and the U.S., under compassionate use. With the recent U.S. approval, we are excited to further expand access to this vital therapy for eligible children with early-onset MLD."
The FDA approval is the culmination of a partnership that started in 2018 with Orchard Therapeutics, which also includes guiding the product through commercial approval by the European Commission (EC) in 2021.
“We congratulate Orchard for reaching this important stage. Our team has had the privilege of supplying every clinical milestone for Lenmeldy and are glad to see it reach FDA approval,” added Luca Alberici, general manager of AGC Biologics Milan.
“I am proud of the work of the Milan team. This demonstrates our unique ability to collaborate on technical processes to deliver groundbreaking treatments to patients worldwide. This approval makes our site one of the rare few in the viral vector and genetically modified cells field that has commercial manufacturing authorization from two of the world’s leading regulatory authorities."
The AGC Biologics Milan site has 30 years of experience in the cell and gene field and expertise with complex advanced cell therapy projects.