Levacetylleucine

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]

^ ^a ^b ^c ^d ^e ^f "Aqneursa- levacetylleucine granule, for suspension". DailyMed. 24 September 2024. Retrieved 5 October 2024.
^ ^a ^b ^c ^d "FDA Approves New Drug to Treat Niemann-Pick Disease, Type C". U.S. Food and Drug Administration (Press release). 24 September 2024. Retrieved 25 September 2024. This article incorporates text from this source, which is in the public domain.
^ "IntraBio Announces U.S. FDA Approval of Aqneursa for the Treatment of Niemann-Pick Disease Type C". IntraBio (Press release). 25 September 2024. Retrieved 26 September 2024.

[Aqneursa_FDA_label-1] ^ ^a ^b ^c ^d ^e ^f "Aqneursa- levacetylleucine granule, for suspension". DailyMed. 24 September 2024. Retrieved 5 October 2024.

[FDA_PR_20240924-2] "FDA Approves New Drug to Treat Niemann-Pick Disease, Type C". U.S. Food and Drug Administration (Press release). 24 September 2024. Retrieved 25 September 2024. This article incorporates text from this source, which is in the public domain.

[3] "IntraBio Announces U.S. FDA Approval of Aqneursa for the Treatment of Niemann-Pick Disease Type C". IntraBio (Press release). 25 September 2024. Retrieved 26 September 2024.

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