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Levacetylleucine
Clinical data
Trade names	Aqneursa
Other names	IB1001
AHFS/Drugs.com	Aqneursa
License data	US DailyMed: Levacetylleucine;
Pregnancy; category	Not recommended;
Routes of; administration	By mouth
ATC code	None;
Legal status
Legal status	US: ℞-only;
Identifiers
	IUPAC name N-Acetyl-L-leucine;
CAS Number	1188-21-2;
PubChem CID	70912;
DrugBank	DB16956;
ChemSpider	1918;
UNII	E915HL7K2O;
KEGG	D12967;
ChEBI	CHEBI:17786;
ChEMBL	ChEMBL56021;
PDB ligand	LAY (PDBe, RCSB PDB);
CompTox Dashboard (EPA)	DTXSID6045870 ;
ECHA InfoCard	100.013.370
Chemical and physical data
Formula	C8H15NO3
Molar mass	173.212 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES O=C(NC(C(=O)O)CC(C)C)C;
	InChI InChI=1S/C8H15NO3/c1-5(2)4-7(8(11)12)9-6(3)10/h5,7H,4H2,1-3H3,(H,9,10)(H,11,12)/t7-/m0/s1; Key:WXNXCEHXYPACJF-ZETCQYMHSA-N;

Levacetylleucine, sold under the brand name Aqneursa, is a medication used for the treatment of neurological manifestations of Niemann-Pick disease type C.^[1]^[2] Levacetylleucine is a modified version of the amino acid leucine (N-Acetyl-L-Leucine).^[1] It is taken by mouth.^[1]

The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.^[1]^[2]

Levacetylleucine was approved for medical use in the United States in September 2024.^[1]^[2]^[3] Levacetylleucine is the second medication approved by the US Food and Drug Administration (FDA) for the treatment of Niemann-Pick disease type C.^[2]

Medical uses

Levacetylleucine is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C in people weighing at least 15 kilograms (33 lb).^[1]^[2]

Adverse effects

The most common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.^[2]

Levacetylleucine may cause embryo-fetal harm if used during pregnancy.^[1]^[2]

History

The safety and efficacy of levacetylleucine for the treatment of Niemann-Pick disease type C were evaluated in a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study.^[2] The duration was twelve weeks for each treatment period.^[2] The study enrolled 60 participants.^[2] To be eligible for the study participants had to be four years of age or older with a confirmed diagnosis of Niemann-Pick disease type C and at least mild disease-related neurological symptoms.^[2] Participants could receive miglustat, an enzyme inhibitor, as background treatment in the study.^[2]

The US Food and Drug Administration (FDA) granted the application for levacetylleucine priority review, fast track, orphan drug, and rare pediatric disease designations.^[2] The FDA granted approval of Aqneursa to IntraBio Inc.^[2]