Levacetylleucine

Last updated

Levacetylleucine
Acetylleucine.svg
Clinical data
Trade names Aqneursa
Other namesIB1001
AHFS/Drugs.com Aqneursa
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 InfoCard 100.013.370 OOjs UI icon edit-ltr-progressive.svg
Chemical and physical data
Formula C8H15NO3
Molar mass 173.212 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 (N-acetyl-L-leucine), 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. [1] It is the L-form of acetylleucine. It is taken by mouth. [1]

Contents

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] The FDA considers it to be a first-in-class medication. [4]

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]

Society and culture

Levacetylleucine was approved for medical use in the United States in September 2024. [1] [2] [5]

Names

Levacetylleucine is the international nonproprietary name. [6]

Research

Levacetylleucine is being studied for the treatment of GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases), [7] ataxia-telangiectasia, [8] Lewy body dementia, [9] amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine. [10]

References

  1. 1 2 3 4 5 6 7 8 9 "Aqneursa- levacetylleucine granule, for suspension". DailyMed. 24 September 2024. Retrieved 5 October 2024.
  2. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 15 "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.[ dead link ]PD-icon.svg 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.
  4. ↑ New Drug Therapy Approvals 2024. U.S. Food and Drug Administration (FDA) (Report). January 2025. Archived from the original (PDF) on 21 January 2025. Retrieved 21 January 2025.
  5. ↑ "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 1 October 2024. Archived from the original on 19 April 2024. Retrieved 29 November 2024.
  6. ↑ World Health Organization (2024). "International nonproprietary names for pharmaceutical substances (INN): proposed INN: list 131". WHO Drug Information. 38 (2). hdl: 10665/378367 . ISBN   9789240098558.
  7. ↑ Martakis K, Claassen J, Gascon-Bayari J, Goldschagg N, Hahn A, Hassan A, et al. (March 2023). "Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses". Neurology. 100 (10): e1072 –e1083. doi:10.1212/WNL.0000000000201660. PMC   9990862 . PMID   36456200.
  8. ↑ Fields T, Patterson M, Bremova-Ertl T, Belcher G, Billington I, Churchill GC, et al. (January 2021). "A master protocol to investigate a novel therapy acetyl-L-leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia". Trials. 22 (1): 84. doi: 10.1186/s13063-020-05009-3 . PMC   7821839 . PMID   33482890.
  9. ↑ Passmore P (15 April 2014). A clinical trial to test amlodipine as a new treatment for vascular dementia. ISRCTN registry (Report). doi: 10.1186/isrctn31208535 .
  10. ↑ Strupp M, Bayer O, Feil K, Straube A (February 2019). "Prophylactic treatment of migraine with and without aura with acetyl-DL-leucine: a case series". Journal of Neurology. 266 (2): 525–529. doi:10.1007/s00415-018-9155-6. PMID   30547273. S2CID   56148131.

Further reading

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