Levacetylleucine

Levacetylleucine
Clinical data
Trade names	Aqneursa
Other names	IB1001
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	C02710;
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]

Society and culture

Legal status

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

Names

Levacetylleucine is the international nonproprietary name.^[4]

Research

N-Acetyl-L-Leucine is being studied for the treatment of GM2 gangliosidoses (Tay-Sachs and Sandhoff diseases)^[5] and ataxia-telangiectasia.^[6]

References

^ ^a ^b ^c ^d ^e ^f ^g ^h ⁱ https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/219132s000lbl.pdf
^ ^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j ^k ^l ^m ⁿ ^o ^p "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.
^ 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.
^ 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.
^ 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.

External links

Clinical trial number NCT05163288 for "A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C" at ClinicalTrials.gov

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[Aqneursa_FDA_label-1] ^ ^a ^b ^c ^d ^e ^f ^g ^h ⁱ https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/219132s000lbl.pdf

[FDA_PR_20240924-2] ^ ^a ^b ^c ^d ^e ^f ^g ^h ⁱ ^j ^k ^l ^m ⁿ ^o ^p "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.

[4] 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.

[5] 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.

[6] 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.

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