1 Recommendations

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

  • for children who have late infantile or early juvenile types, with no clinical signs or symptoms

  • for 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 the company provides atidarsagene autotemcel according to the commercial arrangement.

1.2 Atidarsagene autotemcel should be delivered in a highly specialised service by a specialist multidisciplinary team.

Why the committee made these recommendations

Metachromatic leukodystrophy is a genetic condition that affects the central nervous system. It has a significant effect on the quality of life of children with the condition, and their families and carers. It progresses rapidly, with loss of mobility and cognitive function, and causes early death. Treatment options are limited to managing symptoms and supportive care.

Clinical evidence suggests that the gene therapy atidarsagene autotemcel improves mobility and cognitive function and could correct the enzyme deficiency caused by the condition. But how well atidarsagene autotemcel works in the long term is uncertain.

The cost-effectiveness estimates show that atidarsagene autotemcel provides substantial extra health and quality-of-life benefits. But how much is uncertain, and it varies for the different types of the condition. Taking into account the long-term uncertainty, for children with late infantile and early juvenile forms of the condition, the cost-effectiveness estimates are within what NICE normally considers an acceptable use of NHS resources for highly specialised technologies. So, it is recommended for these children.

  • National Institute for Health and Care Excellence (NICE)