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FDA Grants Approval for Gene Therapy Targeting Pediatric Metachromatic Leukodystrophy

Monday, March 18, 2024

The U.S. Food and Drug Administration has recently approved Lenmeldy (atidarsagene autotemcel) as the first FDA-approved gene therapy for treating children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD).

MLD is a rare genetic disorder that affects the brain and nervous system, caused by a deficiency in the enzyme arylsulfatase A (ARSA). This deficiency leads to the buildup of sulfatides, causing damage to the nervous system, resulting in motor and cognitive function loss and premature death. MLD affects around one in every 40,000 individuals in the United States, with no current cure, and treatment focusing on supportive care.

Dr. Peter Marks, Director of the FDA's Center for Biologics Evaluation and Research (CBER), emphasized the significance of this approval, as it's the first treatment option for children with this rare genetic disease, demonstrating the FDA's commitment to facilitating innovative products to improve patients' lives.

Lenmeldy is a one-time infusion made from the patient's own blood stem cells (HSCs), genetically modified to include functional copies of the ARSA gene. The modified stem cells are transplanted back into the patient, where they produce the ARSA enzyme to break down sulfatides and potentially halt MLD progression. Prior to treatment, patients undergo high-dose chemotherapy to prepare for the modified cell infusion.

Dr. Nicole Verdun, Director of the Office of Therapeutic Products in CBER, highlighted the approval's significance in advancing MLD treatment options, offering hope for improved outcomes and potentially altering disease progression.

Clinical trials evaluating Lenmeldy's safety and efficacy involved 37 children, demonstrating a significant reduction in severe motor impairment or death risk compared to untreated children. Additionally, treated children showed improved survival rates, motor function, and cognitive abilities compared to untreated counterparts.

Common side effects of Lenmeldy include fever, low white blood cell count, mouth sores, respiratory infections, rash, medical line infections, viral infections, gastrointestinal infections, and enlarged liver.

Patients receiving Lenmeldy require monitoring for blood counts and potential complications such as blood clot formation or encephalitis. While there is a potential risk of blood cancer associated with treatment, no cases have been reported in Lenmeldy-treated patients. Lifelong monitoring for hematologic malignancies is recommended.

The application for Lenmeldy received Priority Review, Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy (RMAT) designations. Orchard Therapeutics was granted approval for Lenmeldy by the FDA.



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