Lomitapide

Microsomal Triglyceride Transfer Protein Inhibitor — CARDIOVASCULAR SYSTEM

Key facts

Lomitapide is an ofp drug in the Microsomal Triglyceride Transfer Protein Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2012. Its mechanism of action is classified as Cytochrome P450 3A4 Inhibitors, Microsomal Triglyceride Transfer Protein Inhibitors and P-Glycoprotein Inhibitors. DrugCentral lists 2 molecular targets, including MTR and MTTP. Recorded indications include Familial hypercholesterolemia - homozygous.

• Approval status: ofp (first approved 2012 by the FDA).
• Drug class: Microsomal Triglyceride Transfer Protein Inhibitor.
• Mechanism of action: Cytochrome P450 3A4 Inhibitors; Microsomal Triglyceride Transfer Protein Inhibitors; P-Glycoprotein Inhibitors.
• Molecular targets: MTR and MTTP (2 listed in DrugCentral).
• Indications: Familial hypercholesterolemia - homozygous.
• ATC code: C10AX12 (Other lipid modifying agents).
• Molecular formula: C39H37F6N3O2; molecular weight 693.73 g/mol.

Indications & contraindications

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

Indications (1)

• Familial hypercholesterolemia - homozygous

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 3A4 Inhibitors; Microsomal Triglyceride Transfer Protein Inhibitors; P-Glycoprotein Inhibitors
• Established pharmacologic class: Microsomal Triglyceride Transfer Protein Inhibitor

Molecular targets (2)

Target	Gene	Action	Activity
Methionine synthase	MTR	unknown	IC50 9.30
Microsomal triglyceride transfer protein large subunit	MTTP	inhibitor	IC50 8.10

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2012-12-21	FDA	—	AEGERION	Yes

ATC classification

ATC code	Therapeutic hierarchy
C10AX12	CARDIOVASCULAR SYSTEM › LIPID MODIFYING AGENTS › LIPID MODIFYING AGENTS, PLAIN › Other lipid modifying agents

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: APOB, LDLR, PCSK9 · Testing Required
• FDA label
• HCSC label: LDLR

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
Therapy cessation	2534.4
Weight decreased	1000.7
Diarrhoea	880.6
Flatulence	461.7
Low density lipoprotein increased	427.6
Diet noncompliance	283.2
Nausea	230.8
Product dose omission issue	211.4
Inappropriate schedule of product administration	189.4
Abdominal pain upper	176.9
Alanine aminotransferase increased	167.7
Hepatic steatosis	165.6

Chemical structure

• Molecular formula: C₃₉H₃₇F₆N₃O₂
• Molecular weight: 693.73 g/mol
• CAS number: 182431-12-5
• InChIKey: MBBCVAKAJPKAKM-UHFFFAOYSA-N
• PubChem CID: 9853053

FC(F)(F)CNC(=O)C1(CCCCN2CCC(CC2)NC(=O)C2=C(C=CC=C2)C2=CC=C(C=C2)C(F)(F)F)C2=CC=CC=C2C2=C1C=CC=C2

Frequently asked questions

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

What is Lomitapide used for?

DrugCentral lists the following indications for Lomitapide: Familial hypercholesterolemia - homozygous.

How does Lomitapide work?

Lomitapide is classified as Cytochrome P450 3A4 Inhibitors, Microsomal Triglyceride Transfer Protein Inhibitors and P-Glycoprotein Inhibitors; its molecular targets in DrugCentral include MTR and MTTP (inhibitor).

When was Lomitapide first approved?

Lomitapide was first approved by the FDA in 2012.

What drug class does Lomitapide belong to?

Lomitapide is in the Microsomal Triglyceride Transfer Protein Inhibitor class.

What is the chemical formula of Lomitapide?

Lomitapide has the molecular formula C39H37F6N3O2 and a molecular weight of 693.73 g/mol.

Sources

• DrugCentral drug card 4721 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL2105662 — compound record and bioactivity
• PubChem CID 9853053 — chemical identifiers
• DrugBank DB08827 — drug record
• FDA UNII 82KUB0583F — substance identity
• DailyMed: FDA labels for Lomitapide — current prescribing information (label search)
• UniProt Q99707 (MTR) — target protein
• UniProt P55157 (MTTP) — target protein
• 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.