Levacetylleucine

Levacetylleucine
Clinical data
Trade namesAqneursa
Other namesIB1001
License data
Pregnancy
category
  • Not recommended
Routes of
administration		By mouth
ATC code
  • None
Legal status
Legal status
Identifiers
  • N-Acetyl-L-leucine
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
PDB ligand
CompTox Dashboard (EPA)
ECHA InfoCard100.013.370 Edit this at Wikidata
Chemical and physical data
FormulaC8H15NO3
Molar mass173.212 g·mol−1
3D model (JSmol)
  • O=C(NC(C(=O)O)CC(C)C)C
  • 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]

  1. ^ a b c d e f https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/219132s000lbl.pdf
  2. ^ a b c d e "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. Public Domain 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.