Medical Information

Lomitapide, as a microsomal triglyceride transfer protein (MTP) inhibitor, has significant therapeutic effects on homozygous familial hypercholesterolemia (HoFH), but strict adherence to combination therapy and monitoring of adverse reactions are required. The following provides specific explanations from three aspects: mechanism of action, clinical efficacy, and factors affecting therapeutic efficacy.

1. Mechanism of action

(1) Target inhibition: By specifically inhibiting MTP protein, blocking the assembly of chylomicrons and very low-density lipoprotein (VLDL), plasma low-density lipoprotein cholesterol (LDL-C) levels are reduced.

(2) Metabolic characteristics: Peak concentration reached 6 hours after a single administration, 99.8% bound to plasma proteins, with a half-life of approximately 40 hours, suitable for once daily administration.

2 Clinical effects

(1) Lipid lowering amplitude: Clinical trials have shown that it can reduce LDL-C by 40-50%, and some patients may require LDL separation surgery or other lipid-lowering drugs in combination.

(2) Indication verification: FDA approval based on a key phase III study, HoFH patients showed an average decrease of 50.3% in LDL-C after 28 weeks of treatment.

3 factors affecting therapeutic efficacy

(1) Dose adjustment: The initial dose is 5mg/day, gradually increasing to 60mg/day according to tolerance, and individualized adjustment is required.

(2) Combination therapy: Low fat diet (fat intake<20% total calories) and conventional lipid-lowering regimen must be combined, otherwise the efficacy is limited.

(3) Monitoring requirements: If the transaminase level increases by more than 3 times, medication should be suspended, and those with poor fat absorption should supplement with fat soluble vitamins.