Orchard Therapeutics Achieves Landmark FDA Approval for Lenmeldy™, Pioneering Gene Therapy for Early-onset Metachromatic Leukodystrophy

19 March 2024 | Tuesday | News

Treatment hailed as a breakthrough for children with no prior therapeutic options
Image Source | Public Domain

Image Source | Public Domain

Orchard Therapeutics, now under the stewardship of Kyowa Kirin, announces a historic milestone as the U.S. Food and Drug Administration (FDA) grants approval for Lenmeldy™ (atidarsagene autotemcel). The groundbreaking therapy, previously known as OTL-200, is designated for children with early-onset metachromatic leukodystrophy (MLD), marking a transformative development in pediatric rare disease treatment.

Dr. Bobby Gaspar, Co-founder and CEO of Orchard Therapeutics, expresses profound appreciation for the collaboration of patients and families, stating, "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."

Metachromatic leukodystrophy (MLD) is a devastating genetic disorder that progressively damages the nervous system, resulting in developmental regression and a significantly shortened lifespan. Lenmeldy aims to address the root cause of MLD by integrating functional copies of the human ARSA gene into a patient's hematopoietic stem cells ex vivo, offering a potential one-time treatment with lasting impact.

Dr. Barbara Burton of Ann & Robert H. Lurie Children’s Hospital of Chicago commends the FDA's recognition of Lenmeldy's clinical impact, highlighting the relief it brings to families who have long faced the challenges of this relentless disease.

Maria Kefalas, a parent advocate, emphasizes the urgent need for universal newborn screening for MLD, underscoring the importance of early detection and intervention.

Lenmeldy's approval follows rigorous clinical evaluation, with data from over 12 years of follow-up demonstrating its potential to significantly extend survival and preserve motor and cognitive function in MLD patients.

Orchard Therapeutics will provide further details regarding Lenmeldy's U.S. launch in the coming days.

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