Table 2.

Prescribing summary of lomitapide

Indications and cautions	Adults (≥18 years old) with homozygous familial hypercholesterolemia, if possible with molecular confirmation Not indicated in heterozygous familial hypercholesterolemia patients or other hypercholesterolemic patients Not indicated in children/adolescents <18 years old (lack of evidence) Use with caution in patients >65 years old (limited evidence) For patients with end-stage renal disease with dialysis, do not exceed 40 mg per day; not studied in end-stage renal disease not on dialysis For patients with mild hepatic impairment, do not exceed 40 mg per day; contraindicated in more severe hepatic damage
Dosage	Start with 5 mg once day and uptitrate gradually depending on safety and tolerability at least every 4 weeks. Up to maximum tolerated dose or a maximum of 60 mg per day Dose may be modified according to liver-function tests, tolerability, renal function, and concomitant medication
When?	At least 2 hours after evening meal (empty stomach)
Dietary advice	Low-fat diet, <20% of daily total energy from fat Restriction of alcohol consumption (no more than 1 unit per day)
Supplementation	Vitamin E (400 IU) + linoleic acid (200 mg), ALA (210 mg) + EPA (110 mg) + DHA (80 mg)
Monitoring liver function	Before initiation of treatment Prior to each increase of dose Refer to hepatologist if liver-function testing persists >3×ULN, or data suggest liver steatohepatitis or fibrosis
Hepatic imaging	Screen for liver steatosis, steatohepatitis, and fibrosis at the beginning
Concomitant medication	Moderate and strong CYP3A4 medications and grapefruit juice are contraindicated with lomitapide For patients taking a mild CYP3A4 inhibitor before use of lomitapide, uptitrate the dose according to safety and tolerance If patient requires a mild CYP3A4 inhibitor while taking lomitapide, downtitrate the dose and then uptitrate according to LDL-C reduction, safety and tolerance

Abbreviations: ALA, α-linolenic acid; EPA, eicosapentaenoic acid; DHA, docosahexaenoic acid; ULN, upper limit of normal; LDL-C, low-density-lipoprotein cholesterol.