The National Institute for Health and Care Excellence (NICE) has announced that it does not recommend Orchard Therapeutics’ gene therapy Libmeldy for the treatment of metachromatic leukodystrophy (MLD) in children.
In a statement, NICE said that although clinical trial evidence suggests Libmeldy (autologous CD34+ cells encoding the ARSA gene) – also known as OTL-200 – improves motor and cognitive function and could correct the enzyme deficiency caused by MLD, only short-term evidence is available.
NICE said that, as such, assumptions about the long-term stabilisation of symptoms are ‘very uncertain’ with Libmeldy treatment.
When this uncertainty was taken into account, NICE concluded that the cost-effectiveness estimates for Libmeldy are unlikely to be within the range that it would consider an effective use of NHS resources for highly specialised technologies.
MLD is caused by a deficiency of the enzyme Arylsulfatase-A, which leads to the build-up of sulphatides, eventually destroying the protective myelin sheath of the nervous system. This results in the brain and the peripheral nerves to cease functioning properly.
Patients with the late infantile form of MLD, which is the most common and rapidly progressing type, deteriorate quickly and have a life expectancy of five to eight years old.
Children with early juvenile MLD have a life expectancy of between ten and 20 years after symptoms onset.
Following the NICE rejection, Orchard has proposed a patient access scheme to make Libmeldy available to the NHS with a confidential discount.