28 March 2022 - NICE has published evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children.

Atidarsagene autotemcel is recommended, within its marketing authorisation, as an option for treating metachromatic leukodystrophy with mutations in the arylsulphatase A gene for:

• Children who have late infantile or early juvenile types, with no clinical signs or symptoms
• Children who have the early juvenile type, with early clinical signs or symptoms, and who can still walk independently and have no cognitive decline.

It is recommended only if Orchard Therapeutics provides atidarsagene autotemcel according to the commercial arrangement.

Read NICE highly specialised technologies guidance for atidarsagene autotemcel