acetylleucine

Acetylleucine (N-acetyl-leucine) is a small molecule modified amino acid derivative used for the treatment of various neurological and vestibular disorders. It exists in two primary forms: the racemic mixture N-acetyl-DL-leucine and the purified L-enantiomer N-acetyl-L-leucine (levacetylleucine). The L-enantiomer, marketed under the brand name **Aqneursa**, received FDA approval in September 2024 for the treatment of neurological symptoms in adult and pediatric patients with Niemann-Pick disease type C (NPC). The racemic form, **Tanganil**, has been utilized in France since the 1950s for the symptomatic treatment of vertigo. Acetylleucine is believed to act by stabilizing neuronal membrane potentials and reducing hyperexcitability within the vestibular nucleus. It is also thought to interact with monocarboxylate transporters (specifically MCT1 and MCT2) and may exert neuroprotective effects by improving mitochondrial function and lysosomal homeostasis. Beyond NPC and vertigo, it has been investigated for cerebellar ataxias and nystagmus.

Brand names

AqneursaTanganil

Other names

N-acetyl-leucineN-acetyl-DL-leucineN-acetyl-L-leucinelevacetylleucine