FDA Grants First Approval for Orchard Therapeutics’ Gene Therapy Lenmeldy in Treating Rare Pediatric Neuron Disease

1. The US Food and Drug Administration (FDA) approved Lenmeldy (atidarsagene autotemcel), a gene therapy developed by Orchard Therapeutics, for the treatment of children with metachromatic leukodystrophy (MLD) on March 18, 2024.
2. MLD is a rare genetic disease affecting the brain and nervous system due to deficiency in arylsulfatase A enzyme, leading to progressive neurological deterioration and early death.
3. Lenmeldy is an autologous gene therapy that involves collecting patients' hematopoietic stem cells, modifying them with functional copies of the ARSA gene, and transplanting them back into the patient to produce myeloid cells that break down harmful sulfatides.
4. The FDA approval includes pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile forms of MLD.
5. Prior to receiving Lenmeldy, patients must undergo high-dose chemotherapy to prepare their bone marrow for modified cell infusion.
6. Orchard Therapeutics has already marketed this therapy as Libmeldy in Europe since December 2020.
7. The therapy appears near-curative for some children when administered before symptoms appear or soon after the first signs of disease.
8. Kyowa Kirin, which acquired Orchard Therapeutics in January 2024, will sell the treatment in the U.S..