Posted by Michael Wonder on 18 Mar 2024
FDA approves first gene therapy for children with metachromatic leukodystrophy
18 March 2024 - Today, the US FDA approved Lenmeldy (atidarsagene autotemcel), the first FDA approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy.
The safety and effectiveness of Lenmeldy was assessed based on data from 37 children who received Lenmeldy in two single-arm, open-label clinical trials and in an expanded access program.
