LibmeldyTM is indicated for the treatment of metachromatic leukodystrophy (MLD) characterised by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:1
– In children with late infantile or early juvenile forms, without clinical manifestations of the disease,
– In children with the early juvenile form, with the ability to walk independently and before the onset of cognitive decline*
*Early symptomatic: treatment with Libmeldy of a patient with an early-symptomatic early juvenile form of the disease should be considered:1
– If this patient is able to walk independently, which means that the patient’s GMFC-MLD score is ≤1, and
– If the patient’s cognitive function has not started declining, which means that the patient’s IQ is ≥85.
ARSA: arylsulfatase A; EJ: early juvenile; GMFC: gross motor function classification; HSC: haematopoietic stem cell; IQ: intelligence quotient; MLD: metachromatic leukodystrophy; 0/0: null/null; 0/R: null/residual; R/R: residual/residual
*Based on a systematic literature review of reports from 1921-2006.