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EMA – New treatment for Niemann-Pick type C disease

EMA has recommended granting a marketing authorisation in the European Union (EU) for Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease in adults and children aged six years and older and weighing at least 20 kg. Aqneursa should be used in combination with miglustat, or as a monotherapy in patients where miglustat is not tolerated.

NPC is a rare, progressive, and fatal genetic disorder caused by mutations encoding lysosomal proteins that are essential for the intracellular transport and metabolism of body fats, including cholesterol. Over time, the cells of the central nervous system and the body organs stop working. The course of the disease varies highly depending on the age of onset, but most NPC patients are children and die before the age of 20. There are no curative therapies for NPC. Miglustat is the only medicine authorised to treat NPC and has been shown to slow the general progression of neurological symptoms in patients.

Levacetylleucine is a modified form of the amino acid leucine, which plays a vital role in protein synthesis, muscle growth, and tissue regeneration. Levacetylleucine targets underlying processes of neurological dysfunction. Nonclinical studies have demonstrated that levacetylleucine corrects energy metabolism and improves the production of adenosine triphosphate, the main source of energy for the cerebellar tissues and cells.

More information on the EMA Website.


OpenEdition vous propose de citer ce billet de la manière suivante :
vroby (20 septembre 2025). EMA – New treatment for Niemann-Pick type C disease. ELSIBI. Consulté le 16 mars 2026 à l’adresse https://doi.org/10.58079/14pwl


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