ORTX - Orchard Therapeutics gets positive CHMP opinion for MLD gene therapy

Orchard Therapeutics (ORTX) 11% in premarket on moderate volume, after a positive CHMP opinion recommending approval of Libmeldy, an investigational gene therapy for the treatment of metachromatic leukodystrophy ((MLD)), a rare genetic condition caused by mutations in the ARSA gene, characterized by accumulation of fats called sulfatides, which causes the destruction of the protective fatty layer surrounding the nerves in both the central and the peripheral nervous system.The opinion of the Committee for Medicinal Products for Human Use of the European Medicines Agency will now be reviewed by the European Commission, and final decision is expected by end of this year.If approved, Libmeldy would be the first commercial therapy and first gene therapy for eligible patients with early-onset MLD.

For further details see:

Orchard Therapeutics gets positive CHMP opinion for MLD gene therapy