Libmeldy® 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.
Libmeldy is approved by the European Commission (EC), UK Medicines and Healthcare products Regulatory Agency (MHRA), and the Swiss Agency for Therapeutic Products (Swissmedic). Libmeldy (atidarsagene autotemcel), formerly known as OTL-200, is named Lenmeldy™ in the U.S. and is approved for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early-symptomatic early juvenile metachromatic leukodystrophy (MLD).
Abbreviations
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.
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