National Institute for Health and Care Excellence rejects Orchard Therapeutics’ gene therapy libmeldy

UK – The National Institute for Health and Care Excellence (NICE), has discouraged the use of Orchard Therapeutics’ gene therapy Libmeldy. NICE highlighted that although the clinical trial evidence suggests the drug improves motor and cognitive function and could correct the enzyme deficiency caused by MLD, its long-term stabilization of symptoms was very uncertain. Ortho Therapeutics, a global gene therapy leader, developed Libmeldy for the treatment of metachromatic leukodystrophy (MLD) in children. MLD is a rare hereditary disease characterized by accumulation of fats called sulfatides and causes the destruction of…

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