On March 18, the FDA approved the first gene therapy for children with metachromatic leukodystrophy, a debilitating, rare genetic disease.
The medicine, Lenmeldy (atidarsagene autotemcel), is a one-time infusion made from the patient's hematopoietic stem cells and modified copies of the arylsulfatase A gene, the FDA said. The genetic disease, which is caused by an ARSA gene deficiency, manifests into the loss of motor and cognitive function, and early death.
In a trial, 37 children received Lenmeldy and experienced a significant reduction in the risk of severe motor impairment and death compared to untreated children.
At 5 years old, 71% of treated children could walk without assistance. All study participants who had pre-symptomatic late infantile MLD were alive at 6 years old, compared to 58% of children in the control group.
Before treatment, patients must undergo high-dose chemotherapy. The drug approval was given to Orchard Therapeutics.