Health technology assessment body NICE has welcomed further discussion between Orchard Therapeutics and the National Health Service to resolve its concerns over the company’s one-time gene therapy for the neurodegenerative disorder, metachromatic leukodystrophy, in children.
Orchard Therapeutics has pledged to work to convince NICE about the value of its gene therapy, Libmeldy (atidarsagene autotemcel), after the health technology assessment (HTA) body recommended against making the treatment available on England’s National Health Service for children with metachromatic leukodystrophy (MLD).
In draft guidance, released for public consultation on 9 June, NICE said it recognized that Libmeldy was an “innovative technology...
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