Levacetylleucine

NERVOUS SYSTEM

Key facts

Levacetylleucine is an onp drug catalogued in DrugCentral. It was first approved by the FDA in 2024. Its mechanism of action is classified as Bile Salt Export Pump Inhibitors, Breast Cancer Resistance Protein Inhibitors and Organic Anion Transporter 1 Inhibitors. Recorded indications include Niemann-Pick disease, type C.

• Approval status: onp (first approved 2024 by the FDA).
• Mechanism of action: Bile Salt Export Pump Inhibitors; Breast Cancer Resistance Protein Inhibitors; Organic Anion Transporter 1 Inhibitors.
• Indications: Niemann-Pick disease, type C.
• ATC code: N07XX27 (Other nervous system drugs).
• Molecular formula: C8H15NO3; molecular weight 173.21 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Niemann-Pick disease, type C

Mechanism & targets

• Mechanism of action (MoA): Bile Salt Export Pump Inhibitors; Breast Cancer Resistance Protein Inhibitors; Organic Anion Transporter 1 Inhibitors; Organic Anion Transporter 3 Inhibitors; P-Glycoprotein Inhibitors

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2024-09-24	FDA	—	IntraBio	Yes

ATC classification

ATC code	Therapeutic hierarchy
N07XX27	NERVOUS SYSTEM › OTHER NERVOUS SYSTEM DRUGS › OTHER NERVOUS SYSTEM DRUGS › Other nervous system drugs

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• FDA label: NPC1, NPC2

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Off label use	340.1

Chemical structure

• Molecular formula: C₈H₁₅NO₃
• Molecular weight: 173.21 g/mol
• CAS number: —
• InChIKey: WXNXCEHXYPACJF-ZETCQYMHSA-N
• PubChem CID: 70912

CC(C)C[C@@H](C(=O)O)NC(=O)C

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Levacetylleucine used for?

DrugCentral lists the following indications for Levacetylleucine: Niemann-Pick disease, type C.

How does Levacetylleucine work?

Levacetylleucine is classified as Bile Salt Export Pump Inhibitors, Breast Cancer Resistance Protein Inhibitors and Organic Anion Transporter 1 Inhibitors.

When was Levacetylleucine first approved?

Levacetylleucine was first approved by the FDA in 2024.

What is the chemical formula of Levacetylleucine?

Levacetylleucine has the molecular formula C8H15NO3 and a molecular weight of 173.21 g/mol.

Sources

• DrugCentral drug card 5858 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL56021 — compound record and bioactivity
• PubChem CID 70912 — chemical identifiers
• DrugBank DB16956 — drug record
• FDA UNII E915HL7K2O — substance identity
• DailyMed: FDA labels for Levacetylleucine — current prescribing information (label search)
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.