From Wikipedia, the free encyclopedia
Jump to navigation Jump to search
Lomitapide skeletal.svg
Clinical data
Trade namesJuxtapid (US), Lojuxta (EU)
SynonymsAEGR-773, BMS-201038
License data
  • US: X (Contraindicated)
Routes of
Oral (capsules)
ATC code
Legal status
Legal status
CAS Number
PubChem CID
Chemical and physical data
Molar mass693.719 g/mol
3D model (JSmol)
 ☒N☑Y (what is this?)  (verify)

Lomitapide (INN, marketed as Juxtapid in the US and as Lojuxta in the EU) is a drug used as a lipid-lowering agent for the treatment of familial hypercholesterolemia, developed by Aegerion Pharmaceuticals.[1] It has been tested in clinical trials as single treatment and in combinations with atorvastatin, ezetimibe and fenofibrate.[2][3]

The US Food and Drug Administration (FDA) approved lomitapide on 21 December 2012, as an orphan drug to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL) cholesterol in patients with homozygous familial hypercholesterolemia (HoFH).[4]

On 31 May 2013 the European Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion with a unanimous vote recommending a marketing authorization for lomitapide.[5] On 31 July 2013 the European Commission approved lomitapide as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with HoFH.

Mechanism of action[edit]

Lomitapide inhibits the microsomal triglyceride transfer protein (MTP or MTTP) which is necessary for very low-density lipoprotein (VLDL) assembly and secretion in the liver.[1][6]

On 24 December 2012, drug manufacturer Aegerion announced they had been approved by the FDA to as "an adjunct to a low-fat diet and other lipid-lowering treatments...in patients with homozygous familial hypercholesterolemia (HoFH)."[7][8]

Side effects[edit]

In a Phase III study, lomitapide led to elevated aminotransferase levels and fat accumulation in the liver.[6]


  1. ^ a b H. Spreitzer (12 March 2007). "Neue Wirkstoffe – BMS-201038". Österreichische Apothekerzeitung (in German) (6/2007): 268.
  2. ^ Samaha, Frederick F; James McKenney; LeAnne T Bloedon; William J Sasiela; Daniel J Rader (2008). "Impact of the MTP-Inhibitor, AEGR-733, as Monotherapy and in Combination with Ezetimibe on Lipid Subfractions as Measured by NMR Spectroscopy". Circulation. 118 (5): 469–71. doi:10.1161/CIRCULATIONAHA.108.792689. PMID 18663098.
  3. ^ Aegerion Pharmaceuticals, Inc. Announces AEGR-733 Phase II Data Demonstrates Significant Lowering of LDL Cholesterol with Promising Hepatic Safety Profile Archived 2012-02-29 at the Wayback Machine.
  4. ^ "FDA approves new orphan drug for rare cholesterol disorder"
  5. ^ European Medicines Agency: Lojuxta
  6. ^ a b Cuchel, M.; Bloedon, L. T.; Szapary, P. O.; Kolansky, D. M.; Wolfe, M. L.; Sarkis, A.; Millar, J. S.; Ikewaki, K.; Siegelman, E. S.; Gregg, R. E.; Rader, D. J. (2007). "Inhibition of Microsomal Triglyceride Transfer Protein in Familial Hypercholesterolemia". New England Journal of Medicine. 356 (2): 148–156. doi:10.1056/NEJMoa061189. PMID 17215532.
  7. ^ "FDA Approves Juxtapid for Homozygous Familial Hypercholesteolemia". 26 December 2012.
  8. ^ "FDA Approves Aegerion Pharmaceuticals' Juxtapid (lomitapide) Capsules for Homozygous Familial Hypercholesterolemia (HoFH)" (Press release). Aegerion Pharmaceuticals. 24 December 2012.

External links[edit]