AGC Biologics Confirms Cell and Gene Therapy Commercial Expertise as Manufacturer of Orchard Therapeutics’ Newly Approved Libmeldy

AGC Biologics, a leading global Biopharmaceutical Contract Development and Manufacturing Organization (CDMO), is the first manufacturer of Orchard Therapeutics’ Libmeldy, which was recently approved by the European Commission (EC) as a one-time therapy for eligible patients with early-onset Metachromatic Leukodystrophy (MLD).

The EC has granted full (standard) market authorization for Libmeldy™ (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy manufactured at AGC Biologics’ Milan facility. Orchard Therapeutics is already underway with EU launch preparations to support commercial-scale drug manufacturing at the AGC Biologics Milan facility.

“AGC Biologics is delighted to have successfully partnered with Orchard Therapeutics on this great milestone,” says AGC Biologics Chief Business Officer, Mark Womack. “We’re very proud to be one of few Cell and Gene Therapy CDMOs with commercial experience – Libmeldy being the third commercial product we’ve manufactured – and we look forward to continuing our partnership with Orchard to provide this life-changing therapy to those affected by MLD.”

“We are very honored to have been a part of the entire clinical journey of this product, from the very first treated patient, all the way to market approval. As manufacturer of both lentiviral vector and drug product, we are proud to support Orchard in treating this devastating disease,” says AGC Biologics General Manager of Milan, Luca Alberici.

“The EC approval of Libmeldy opens up tremendous possibilities for eligible MLD children faced with this devastating disease,” says Bobby Gaspar, M.D., Ph.D., Chief Executive Officer of Orchard. “We are humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU, and confident in the capabilities of our partners at AGC Biologics to help us achieve this goal.”

MLD is a rare neurodegenerative disorder caused by mutations in the ARSA gene. While there are several forms of MLD, the disorder primarily affects young children and disease progression and life expectancy varies based on age of symptom onset. Libmeldy is designed to correct the genetic cause of MLD by inserting functional copies of the ARSA gene into the genome of a patient’s own hematopoietic stem cells (HSCs) using a self-inactivating (SIN) lentiviral vector. It is approved in the EU for children with i) late infantile or early juvenile forms, without clinical manifestations of the disease, or ii) the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline. Libmeldy™ is the first therapy approved for eligible patients with early-onset MLD.