FDA BLA approval: Lenmeldy (Catapult’s regulatory round-up – March 2024)
The FDA has 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 (MLD). Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene.Please find further information here.
Published on the 2nd of April 2024 in Catapult’s regulatory round-up for March 2024.
OpenEdition vous propose de citer ce billet de la manière suivante :
adelage (5 novembre 2024). FDA BLA approval: Lenmeldy (Catapult’s regulatory round-up – March 2024). ELSIBI. Consulté le 16 janvier 2025 à l’adresse https://doi.org/10.58079/12mit
