NICE issues final evaluation determination on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy
DRAFT guidance recommends this gene therapy medicinal product that expresses the human ARSA gene as an option for treating some children with the rare, life-limiting inherited neurodegenerative condition metachromatic leukodystrophy with mutations in the ARSA gene.
Source:
National Institute for Health and Care Excellence
SPS commentary:
A related press release notes that this treatment, given as a single-dose IV infusion, is the most expensive that NICE has ever evaluated (£2.8 million list price). There are around 5 children born each year with metachromatic leukodystrophy, which causes severe damage to the nervous system and organs, and is associated with a life expectancy of 5-8 years.
A press release from NHS England notes that the treatment will be delivered by a specialist service and will be available on the NHS from Royal Manchester’s Children Hospital in collaboration with Manchester’s Centre for Genomic Medicine at Saint Mary’s Hospital. This is the only site in the UK that will administer the treatment.