Lomitapide, a microsomal triglyceride transfer protein inhibitor for the treatment of hypercholesterolemia

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New lipid-lowering agents include microsomal triglyceride transfer protein (MTP) inhibitors, which may have a role in the treatment of hypercholesterolemia. Clinical applications of MTP inhibitors have been focused primarily on high-dose monotherapy to produce substantial reductions in LDL-cholesterol levels (particularly for patients with homozygous familial hypercholesterolemia). However, this strategy has been associated with a high rate and severity of gastrointestinal and hepatic adverse events that has prohibited the use of these agents. Data suggest the LDL-cholesterol-lowering efficacy of low-dose lomitapide (AEGR-733, formerly BMS-201038), under development by Aegerion Pharmaceuticals Inc, in patients with familial hypercholesterolemia, both as a single agent and in combination with commonly prescribed lipid-lowering therapies. MTP inhibition with lomitapide may offer a treatment option for patients who cannot tolerate statin therapy or who experience insufficient LDL-cholesterol reduction with available therapies. However, the safety concerns for MTP inhibitors for the treatment of hyperlipidemia must be fully addressed, and the assessment of the risk-to-benefit ratio for MTP inhibitors in patients at different levels of cardiovascular-disease risk is required before clinical use of this class of drugs may be recommended. © Thomson Reuters (Scientific) Ltd.

Author-supplied keywords

  • IC50
  • NCT00359281
  • NCT00405067
  • NCT00474240
  • NCT00559962
  • NCT00690443
  • NCT00730236
  • NCT00943306
  • aegr 733
  • area under the curve
  • atorvastatin
  • clinical trial
  • controlled clinical trial
  • dextromethorphan
  • drug dose escalation
  • drug synthesis
  • drug withdrawal
  • ezetimibe
  • familial hypercholesterolemia
  • fenofibrate
  • gastrointestinal disease
  • human
  • infection
  • lomitapide
  • maximum plasma concentration
  • microsomal triglyceride transfer protein inhibitor
  • musculoskeletal disease
  • nicotinic acid
  • nonhuman
  • patent
  • randomized controlled trial
  • review
  • rosuvastatin
  • side effect
  • simvastatin
  • triacylglycerol
  • triacylglycerol blood level
  • unclassified drug

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  • Rizzo M.

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