Orchard Therapeutics: European Medicines Approves Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)

   European Commission (EC) granted full (standard) market authorization for Libmeldy (autologous CD34+ cells encoding the ARSA gene), a lentiviral vector-based gene therapy approved for the treatment of metachromatic leukodystrophy (MLD), characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity in 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. 

MLD is a very rare, fatal genetic disorder caused by mutations in the ARSA gene which lead to neurological damage and developmental regression. In its most severe and common forms, young children rapidly lose the ability to walk, talk, and interact with the world around them, and most pass away before adolescence.

Libmeldy becomes the first therapy approved for eligible patients with early-onset MLD. Libmeldy is designed as a one-time therapy that aims to correct the underlying genetic cause of MLD, offering eligible young patients the potential for long-term positive effects on cognitive development and maintenance of motor function at ages at which untreated patients show severe motor and cognitive impairments.

“Today’s EC approval of Libmeldy opens up tremendous new possibilities for eligible MLD children faced with this devastating disease where previously no approved treatment options existed,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. “Libmeldy is Orchard’s first product approval as a company, and I am extremely proud of the entire team who helped achieve this milestone. We are grateful for and humbled by the opportunity to bring this remarkable innovation to young eligible patients in the EU.”

Source: https://www.globenewswire.com/news-release/2020/12/21/2148486/0/en/Orchard-Therapeutics-Receives-EC-Approval-for-Libmeldy-for-the-Treatment-of-Early-Onset-Metachromatic-Leukodystrophy-MLD.html