FDA Approves First Gene Therapy to Treat Children with Metachromatic Leukodystrophy

On March 18, 2024, the FDA approved Orchard Therapeutics’ LENMELDY, the first approved gene therapy for the treatment of children with metachromatic leukodysrophy (MLD).  MLD is a rare, fatal genetic disease affecting approximately one in every 40,000 individuals in the U.S.  LENMELDY is made from patient’s own hematopoietic (blood) stem cells, and the genetically modified stem cells are transplanted back into the patient as a one-time, single-dose infusion.  Bobby Gaspar, M.D., Ph.D., co-founder and CEO of Orchard Therapeutics stated, “The FDA approval of Lenmeldy opens up tremendous new possibilities for children in the U.S. with early-onset MLD who previously had no treatment options beyond supportive and end-of-life care.”