9 July 2021 - OTL-200 (Libmeldy) is a gene therapy medicinal product that expresses the human ARSA gene.

OTL-200 is not recommended, within its marketing authorisation, for the treatment of children with metachromatic leukodystrophy characterised by biallelic mutations in the arylsulphatase A (ARSA) gene that reduce ARSA enzyme activity who have:

• Late infantile or early juvenile types, with no clinical signs or symptoms
• The early juvenile type, with early clinical signs or symptoms, and who can still walk independently and have no cognitive decline

Read NICE evaluation consultation document