Levacetylleucine is a modified form of leucine used to treat neurological symptoms in patients with Niemann-Pick disease type C. Levacetylleucine (L-acetylleucine) is a modified acetylated derivative of the amino acid leucine.
- Mechanism
- Curator reviewed · 5 references
Following oral administration, levacetylleucine is taken up via ubiquitously expressed monocarboxylate transporters and distributed to all body tissues. It enters enzyme-controlled pathways that correct metabolic dysfunction and improves adenosine triphosphate (ATP) energy production. The normalization of energy metabolism ameliorates mitochondrial and lysosomal dysfunction and leads to a reduction in the storage of unesterified cholesterol and sphingolipids. Levacetylleucine has also been shown to normalize neuronal membrane potentials in a guinea pig model, thereby improving cellular signaling processes and restoring and protecting neuronal circuits.
The specific molecular target of levacetylleucine in the context of Niemann-Pick disease type C (NPC) is unknown.
- Primary indication
- Levacetylleucine is indicated for the treatment of neurological symptoms of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.Curator reviewed · 2 structured indications
- Formula / weight
- C8H15NO3 · 173.212 g/mol (avg)
- First approval
- United States, 2024 · European Union, 2026
- Also known as
- (s)-2-acetamido-4-methylpentanoic acid · Acetyl-l-leucine · Acetylleucine, (s)- · Acetylleucine, l- · L-Acetylleucine
- Code names
- NSC-206316
- Brand names
Resolves to
WXNXCEHXYPACJF-ZETCQYMHSA-NSMILESCC(C)C[C@H](NC(C)=O)C(O)=OWhat you can answer from here — as of September 24, 2026
Which other approved drugs treat the same conditions as Levacetylleucine, and which companies have late-stage candidates in those indications?
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