Repositioning Candidate Details
| Candidate ID: | R1162 |
| Source ID: | DB08827 |
| Source Type: | approved; investigational |
| Compound Type: | small molecule |
| Compound Name: | Lomitapide |
| Synonyms: | -- |
| Molecular Formula: | C39H37F6N3O2 |
| SMILES: | 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=CC=CC=C12 |
| Structure: |
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| DrugBank Description: | Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor used in homozygous familial hypercholesterolemia (HoFH) patients. It is marketed under the name Juxtapid (R). |
| CAS Number: | 182431-12-5 |
| Molecular Weight: | 693.7204 |
| DrugBank Indication: | Used in homozygous familial hypercholesterolemia (HoFH) patients to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C). |
| DrugBank Pharmacology: | Lomitapide directly inhibits microsomal triglyceride transfer protein (MTP). |
| DrugBank MoA: | Within the lumen of the endoplasmic reticulum, lomitapide inhibits microsomal triglyceride transfer protein (MTP), which prevents the formation of apolipoprotein B, and, thus, the formation of VLDL and chylomicrons as well. Altogether, this leads to a reduction of low-density lipoprotein cholesterol. |
| Targets: | Microsomal triglyceride transfer protein large subunit antagonist |
| Inclusion Criteria: | Indication associated |
| Strategy ID | Strategy | Synonyms | Related Targets | Related Drugs |
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| Target ID | Target Name | GENE | Action | Class | UniProtKB ID | Entry Name |
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