Inhibition of hepatic microsomal triglyceride transfer protein – a novel therapeutic option for treatment of homozygous familial hypercholesterolemia

Figures & data

Table 1 Trials of cholesterol lowering with lomitapide in homozygous familial hypercholesterolemia

Study type	Number of patients, male/female	Age of patients (years)	CVD symptoms	Medication (inhibition of MTTP)	Dose	Duration	Decrease in LDLC	Adverse events	Study (ref)
Open-label, single-arm	3/3	18–40	2	AEGR 733 (lomitapide)	Final 1.0 mg/kg/day	16 weeks	−50.9% (dose 1.0 mg/kg/day)	Elevation of liver transaminase levels and accumulation of hepatic fat	^Citation63
Open-label, single-arm	16/13	18–55	27	AEGR 733 (lomitapide)	Median 40 mg/day	Efficacy phase 26 weeks (weeks 1–26) Safety phase 52 weeks (weeks 26–78)	−50% (week 56) −38% (week 78)	Most frequent were gastrointestinal symptoms and elevation of liver transaminase levels	^Citation64

Abbreviations: CVD, cardiovascular disease; MTTP, microsomal triglyceride transfer protein; LDLC, low-density lipoprotein cholesterol; ref, reference.

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