Pharma Focus Asia

AGC Biologics confirms commercial expertise in gene and cell therapy as manufacturer of Libmeldy

Tuesday, January 05, 2021

AGC Biologics, a prominent leading pharmaceutical contract development and manufacturing organization (CDMO), is the first manufacturer of Orchard Therapeutics' Libmeldy ™ product, recently approved by the European Commission (CE) as a one-time therapy for valid patients with primary-onset metachromatic leukodystophy (MLD).

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

"AGC Biologics is pleased to have partnered with Orchard Therapeutics on this great milestone," said AGC Biologics Chief Business Officer Mark Womack . "We are very proud to be one of the few gene and cell therapy CDMOs with commercial experience - Libmeldy ™ being the third commercial product we have manufactured - and we can't wait to continue our partnership with Orchard to provide this game-changing therapy. of life to all those affected by MLD. "

"It is a great honor to have been a part of the entire clinical journey of this product, from the first patient treated, all the way to market approval. As a manufacturer of the lentiviral vector and drug product, we are proud to support Orchard in the treatment of this devastating disease, "explained the general manager of AGC Biologics in Milan, Luca Alberici .

"The EC approval of Libmeldy opens up enormous possibilities for children with valid MLDs who are facing this devastating disease," said Bobby Gaspar, MD, Ph.D. and CEO of Orchard. "It is an honor to have the opportunity to bring this outstanding innovation to selected young patients from the EU, and we are confident in the capabilities of our partners at AGC Biologics to help us achieve this goal."

MLD is a rare neurodegenerative disease caused by mutations in the ARSA gene . Although there are several forms of MLD, the disease mainly affects young people and the progression of the disease and life expectancy varies according to the age of onset of symptoms. Libmeldy ™ is designed to correct the genetic cause of MLD by inserting working copies of the ARSA gene in the genome of the patient's own hematopoietic stem cells (HSCs) using the self-inactivating lentiviral vector (SIN). It is approved in the EU for children with i) primary infantile or juvenile late forms, without clinical manifestations of the disease, or ii) primary juvenile form, with initial clinical manifestations of the disease, who continue to have the ability to walk independently and before the onset of cognitive decline. Libmeldy ™ is the first approved therapy for selected patients with primary-onset MLD.

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