AGC Biologics, a global biopharmaceutical CDMO, today announced that the U.S. FDA has cleared its 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 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 R&D stages to this DP commercial milestone. 

“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,” said Patricio Massera, CEO of AGC Biologics.  

Luca Alberici, General Manager of AGC Biologics Milan, said, “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.”