Indication
Treatment of metachromatic leukodystrophy characterized by biallelic mutations in the 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
Medicine details
- Medicine name:
- atidarsagene autotemcel (Libmeldy)
- SMC ID:
- SMC2886
- Pharmaceutical company
- Orchard Therapeutics
- BNF chapter
- Nutrition and blood
- Submission type
- Ultra-orphan reassessment
- Publication due date:
- TBC
- SMC meeting date:
- TBC
- Patient group submission deadline:
- TBC