Indication

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 the pre-symptomatic late infantile (PSLI) or pre-symptomatic early juvenile (PSEJ) forms 

– In children with the early symptomatic early juvenile (ESEJ) form, who still have 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 (Gross Motor Function Classification for Metachromatic Leukodystrophy) score is ≤ 1, and 

– If the patient’s cognitive function is not rapidly declining, and 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).