NICE Endorses High-Priced Gene Therapy for Rare Neurodegenerative Disorder in Children
The UK’s National Institute for Health and Care Excellence (NICE) has recommended that the National Health Service (NHS) offer Orchard Therapeutics’ costly gene therapy, Libmeldy (atidarsagene autotemcel), for children with a rare, inherited neurodegenerative condition called metachromatic leukodystrophy (MLD).
NICE said that the drug, which has a list price of approximately $3.79 million, is the most expensive the cost regulator has ever evaluated. But NHS patients will pay a confidential discounted price that NICE negotiated with the drugmaker.
Administered via a single-dose infusion, Libmedly is the first therapy approved in the UK to treat MLD which corrects the genetic cause of the disease by substituting functional copies of the faulty genes into the patient’s stem cells.