Orchard Therapeutics PLC (ORTX)

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Orchard Therapeutics Gets CHMP Approval for Libmeldy
6:29 am ET October 16, 2020 (Dow Jones) Print

By Chris Wack

Orchard Therapeutics Plc said Friday the Committee for Medicinal Products for Human Use of the European Medicines Agency has adopted a positive opinion recommending full, or standard, marketing authorization for Libmeldy, an investigational gene therapy for the treatment of metachromatic leukodystrophy.

The company said the CHMP's positive opinion will now be reviewed by the European Commission, which has the authority to grant marketing authorization for Libmeldy in the European Union.

A final decision by the EC for Libmeldy is anticipated before the end of 2020. If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

MLD is a severe genetic condition caused by mutations in the ARSA gene which lead to neurological damage and developmental regression.

Libmeldy is designed as a one-time gene therapy. The positive CHMP opinion is supported by clinical studies of Libmeldy in both pre- and early- symptomatic, early-onset MLD patients. Clinical efficacy was based on the integrated analysis of results from 29 patients with early-onset MLD who were all treated with Libmeldy prepared as a fresh formulation.