levacetylleucine

Levacetylleucine (L-acetylleucine, N-acetyl-L-leucine) is a modified acetylated derivative of the amino acid leucine. It is used for the treatment of neurological manifestations in patients with Niemann-Pick disease type C (NPC), a rare genetic disorder characterized by impaired lipid metabolism and neurodegeneration. The drug was approved in the United States in September 2024 under the brand name Aqneursa for use in individuals weighing at least 15 kg with NPC[1][3]. Levacetylleucine acts as a small molecule that enters cells via monocarboxylate transporters and participates in metabolic pathways to correct energy dysfunctions. Its mechanism includes improving ATP production, normalizing mitochondrial and lysosomal function, reducing storage of unesterified cholesterol and sphingolipids, and restoring neuronal membrane potentials[1][5]. While its precise molecular target remains unknown[1], it has been shown to ameliorate ataxia symptoms such as balance and coordination problems associated with NPC[5].

Brand names

Aqneursa

Other names

levacetylleucineN-acetyl-L-leucineL-acetylleucine