Pediatric Neuron Disease Gets Its First Gene Therapy

MedPage Today March 18, 2024
Judy George

— FDA approves arsa-cel for metachromatic leukodystrophy

The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), the agency announced on Monday.

“This is the first FDA-approved treatment option for children who have this rare genetic disease,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.

MLD is caused by a mutation in the ARSA gene that results in sulfatides accumulating in the central nervous system and peripheral nervous system, leading to progressive dysmyelination, neuroinflammation, and neurodegeneration. This causes loss of motor and cognitive functions and, ultimately, death. There is no cure...

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