AGC Biologics To Manufacture First-Ever FDA Approved Gene Therapy For Early-Onset Metachromatic Leukodystrophy, Orchard Therapeutics' Lenmeldy™

AGC Biologics received U.S. Food and Drug Administration (FDA) clearance at the company’s Milan site to begin commercial manufacturing for the lentiviral vector and genetically modified cell drug product (DP) of Orchard Therapeutics’ Lenmeldy™ (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy for the treatment of pre-symptomatic late infantile, pre-symptomatic early juvenile and early symptomatic early juvenile metachromatic leukodystrophy (MLD). Lenmeldy received approval by the FDA on Monday, March 18.

Lenmeldy is the first therapy to receive U.S. FDA approval for MLD. With this announcement, AGC Biologics Milan becomes the only global CDMO site to receive the regulatory agency’s clearance to produce this treatment for patients in the U.S. commercially. 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. AGC Biologics’ Milan site is uniquely connected to this product’s full lifecycle, as the facility and its scientific teams partnered with each company that owned this treatment’s IP over the last 20 years to help advance it from research and development stages to this DP commercial milestone.

“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,” said 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."

"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 U.S.," said Nicoletta Loggia, Ph.D., 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 AGC Biologics Milan location has 30 years of experience in the cell and gene field and expertise with complex advanced cell therapy projects. The core team has guided three cell therapy products from development to commercial stages and has manufactured hundreds of batches of cell therapies for clinical and commercial usage. The site serves as the AGC Biologics’ Global Cell and Gene Center of Excellence for AGC Biologics.

The team has a track record for helping developers meet regulatory guidelines and achieve quality performance metrics, and experience with navigating the unique complexities of technology transfers and scaling up and scaling out manufacturing within the cell and gene space.