levacetylleucine

Levacetylleucine is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C in people weighing at least {{convert|15|kg|lb}}.

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

Levacetylleucine may cause embryo-fetal harm if used during pregnancy.

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. The duration was twelve weeks for each treatment period. The study enrolled 60 participants. 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. Participants could receive miglustat, an enzyme inhibitor, as background treatment in the study. The trial was conducted at eleven sites in seven countries in Europe and Australia.{{cite web | title=Drug Trials Snapshots: Aqneursa | website=U.S. Food and Drug Administration (FDA) | date=24 September 2024 | url=https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-aqneursa | access-date=2 July 2025}} {{PD-notice}}

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

Levacetylleucine was approved for medical use in the United States in September 2024.{{cite web | title=Novel Drug Approvals for 2024 | website=U.S. Food and Drug Administration (FDA) | date=1 October 2024 | url=https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024 | archive-url=https://web.archive.org/web/20240419180642/https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024 | url-status=live | archive-date=19 April 2024 | access-date=29 November 2024}}

Levacetylleucine is the international nonproprietary name.{{cite journal | vauthors = ((World Health Organization)) | year = 2025 | title = International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 93 | journal = WHO Drug Information | volume = 39 | issue = 1 | hdl = 10665/381075 | hdl-access = free | author-link = World Health Organization }}

Levacetylleucine is being studied for the treatment of GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases),{{cite journal | vauthors = Martakis K, Claassen J, Gascon-Bayari J, Goldschagg N, Hahn A, Hassan A, Hennig A, Jones S, Kay R, Lau H, Perlman S, Sharma R, Schneider S, Bremova-Ertl T | title = Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses | journal = Neurology | volume = 100 | issue = 10 | pages = e1072–e1083 | date = March 2023 | pmid = 36456200 | pmc = 9990862 | doi = 10.1212/WNL.0000000000201660 }} ataxia-telangiectasia,{{cite journal | vauthors = Fields T, Patterson M, Bremova-Ertl T, Belcher G, Billington I, Churchill GC, Davis W, Evans W, Flint S, Galione A, Granzer U, Greenfield J, Karl R, Kay R, Lewi D, Mathieson T, Meyer T, Pangonis D, Platt FM, Tsang L, Verburg C, Factor M, Strupp M | title = 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 | journal = Trials | volume = 22 | issue = 1 | pages = 84 | date = January 2021 | pmid = 33482890 | pmc = 7821839 | doi = 10.1186/s13063-020-05009-3 | doi-access = free }} Lewy body dementia,{{Cite report | vauthors = Passmore P |date=15 April 2014 |title=A clinical trial to test amlodipine as a new treatment for vascular dementia | work = ISRCTN registry |doi=10.1186/isrctn31208535 |doi-access=free}} amyotrophic lateral sclerosis, restless legs syndrome, multiple sclerosis, and migraine.{{cite journal | vauthors = Strupp M, Bayer O, Feil K, Straube A | title = Prophylactic treatment of migraine with and without aura with acetyl-DL-leucine: a case series | journal = Journal of Neurology | volume = 266 | issue = 2 | pages = 525–529 | date = February 2019 | pmid = 30547273 | doi = 10.1007/s00415-018-9155-6 | s2cid = 56148131 }}

{{reflist}}

{{refbegin}}

• {{cite journal | vauthors = Churchill GC, Strupp M, Factor C, Bremova-Ertl T, Factor M, Patterson MC, Platt FM, Galione A | title = Acetylation turns leucine into a drug by membrane transporter switching | journal = Scientific Reports | volume = 11 | issue = 1 | pages = 15812 | date = August 2021 | pmid = 34349180 | pmc = 8338929 | doi = 10.1038/s41598-021-95255-5 | bibcode = 2021NatSR..1115812C }}
• {{cite journal | vauthors = Bremova-Ertl T, Ramaswami U, Brands M, Foltan T, Gautschi M, Gissen P, Gowing F, Hahn A, Jones S, Kay R, Kolnikova M, Arash-Kaps L, Marquardt T, Mengel E, Park JH, Reichmannová S, Schneider SA, Sivananthan S, Walterfang M, Wibawa P, Strupp M, Martakis K | title = Trial of N-Acetyl-l-Leucine in Niemann-Pick Disease Type C | journal = The New England Journal of Medicine | volume = 390 | issue = 5 | pages = 421–431 | date = February 2024 | pmid = 38294974 | doi = 10.1056/NEJMoa2310151 }}
• {{cite journal | vauthors = Tifft CJ | title = N-Acetyl-l-Leucine and Neurodegenerative Disease | journal = The New England Journal of Medicine | volume = 390 | issue = 5 | pages = 467–470 | date = February 2024 | pmid = 38294981 | doi = 10.1056/NEJMe2313791 }}

{{refend}}

• {{ClinicalTrialsGov|NCT05163288|A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C}}

{{Portal bar | Medicine}}

{{Authority control}}

Category:Amides

Category:Amino acids

Category:Orphan drugs