Approval Beckons For Orchard’s Gene Therapy Libmeldy

Orchard Therapeutics is on the cusp of the first approval for its ex vivo gene therapy, Libmeldy, for metachromatic leukodystrophy.

Orchard Therapeutics Limited is hoping to gain the first approval by the end of the year for its one-time gene therapy for metachromatic leukodystrophy (MLD), Libmeldy (OTL-2000), in the EU. A launch is expected to follow in early 2021.

Libmeldy (cryopreserved autologous CD34+ cells encoding the arylsulfatase-A, or ARSA, gene) has been granted a positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) for full marketing authorization for

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