NICE rejects Orchard’s gene therapy for rare childhood disease MLD

Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.

The agency said that while there was evidence of a short-term benefit with Libmeldy (atidarsagene autotemcel or OTL-200), assumptions about its long-term effects are uncertain, making it unclear whether it will offer value for money to the NHS.

The decision kicks off a comment period and further negotiations between the company, NICE and NHS England on the price of Libmeldy. Orchard has already offered a confidential discount on the therapy.

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