NICE Rejects Gene Therapy for Treating Metachromatic Leukodystrophy in Children

The UK’s National Institute for Health and Care Excellence (NICE) has recommended that the National Health Service (NHS) not offer Orchard Therapeutics’ gene therapy Libmeldy for treating children with metachromatic leukodystrophy (MLD).

MLD is a rare genetic disorder spurred by an enzyme deficiency that causes excessive lipid buildup in cells, including in the brain, spinal cord and peripheral nerves.

Although Libmedly (autologous CD34+ cells encoding the ARSA gene) showed it could boost motor and cognitive function and fix the enzyme deficiency, only short-term trial data are currently available, NICE said.  

The agency said it couldn’t justify the therapy’s cost for NHS use given the limited supporting data.