atidarsagene autotemcel (Libmeldy)

following a submission through the ultra-orphan framework:

atidarsagene autotemcel (Libmeldy®) is accepted for use within NHSScotland.

Indication under review: Treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:

• in children with late infantile or early juvenile forms, without clinical manifestations of the disease
• in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

In combined data from phase I/II studies and three early access programmes, atidarsagene autotemcel increased gross motor function scores in pre-symptomatic late infantile and in pre- and early symptomatic early juvenile MLD patients, when compared with a natural history cohort of patients with MLD.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.

This advice applies only in the context of approved NHSScotland Patient Access Scheme (PAS) arrangements delivering the cost-effectiveness results upon which the decision was based, or PAS/ list prices that are equivalent or lower.