atidarsagene autotemcel (Libmeldy®)

Status: Excluded due to NICE appraisal
Product meets AWMSG exclusion criteria due to NICE appraisal HST18: Atidarsagene autotemcel for treating metachromatic leukodystrophy
Medicine details
Medicine name	atidarsagene autotemcel (Libmeldy®)
Formulation	single dose intravenous infusion
Reference number	4156
Indication	Treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase 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.
Company	Orchard Therapeutics
BNF chapter	Central nervous system
Submission type	N/A
Status	Excluded due to NICE appraisal
Date of issue	04/02/2022
NICE guidance	HST18: Atidarsagene autotemcel for treating metachromatic leukodystrophy