Last reviewed 2026-04-20 · How we verify

Juxtapid (LOMITAPIDE)

Juxtapid works by blocking the protein that helps transport cholesterol into the bloodstream.

Juxtapid (LOMITAPIDE) is a microsomal triglyceride transfer protein inhibitor, a small molecule drug developed by Aegerion and currently owned by Chiesi. It targets the microsomal triglyceride transfer protein large subunit to treat familial hypercholesterolemia in homozygous patients. Juxtapid was FDA-approved in 2012 and remains a patented product with no generic manufacturers. Key safety considerations include its low bioavailability and long half-life. It is used to reduce cholesterol levels in patients with severe genetic cholesterol disorders.

At a glance

Mechanism of action

JUXTAPID directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endoplasmic reticulum, thereby preventing the assembly of apo B-containing lipoproteins in enterocytes and hepatocytes. This inhibits the synthesis of chylomicrons and VLDL. The inhibition of the synthesis of VLDL leads to reduced levels of plasma LDL-C.

Approved indications

• Familial hypercholesterolemia - homozygous

Boxed warnings

• WARNING: RISK OF HEPATOTOXICITY JUXTAPID can cause elevations in transaminases. In the adult clinical trial, 10 (34%) of the 29 patients treated with JUXTAPID had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3 times the upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase. In the pediatric clinical trial (5 to 17 years of age), 6 (14%) of the 43 patients experienced elevations in ALT and/or AST ≥ 3 times ULN. No concomitant clinically meaningful elevations in total bilirubin or alkaline phosphatase were observed [see Warnings and Precautions (5.1) ]. JUXTAPID also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat in adult patients was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy (MRS). The median absolute increase in hepatic fat in pediatric patients aged 5 to 17 years was 4% after 24 weeks and 104 weeks of treatment, from 3% at baseline, measured by nuclear magnetic resonance (NMR). Hepatic steatosis associated with JUXTAPID treatment may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis [see Warnings and Precautions (5.1) ]. Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment and then ALT and AST regularly as recommended. During treatment, adjust the dose of JUXTAPID if the ALT or AST are ≥3 times ULN. Discontinue JUXTAPID for clinically significant liver toxicity [ see Dosage and Administration (2.7) and Warnings and Precautions (5.1) ]. Because of the risk of hepatotoxicity, JUXTAPID is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the JUXTAPID REMS Program [see Warnings and Precautions (5.2) ]. Prescribe JUXTAPID only to patients with a clinical or laboratory diagnosis consistent with HoFH. The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH [see Indications and Usage (1) ] . WARNING: RISK OF HEPATOTOXICITY See full prescribing information for complete boxed warning. JUXTAPID can cause elevations in transaminases ( 5.1 ). Measure alanine and aspartate aminotransferases (ALT, AST), alkaline phosphatase, and total bilirubin before initiating treatment and then ALT and AST regularly as recommended ( 2.5 , 5.1 ). During treatment, adjust the dose of JUXTAPID if the ALT or AST is ≥3 times the upper limit of normal (ULN) ( 2.5 , 5.1 ). Discontinue JUXTAPID for clinically significant liver toxicity ( 2.5 , 5.1 ). JUXTAPID increases hepatic fat (hepatic steatosis) with or without concomitant increases in transaminases ( 5.1 ). Hepatic steatosis associated with JUXTAPID may be a risk factor for progressive liver disease, including steatohepatitis and cirrhosis ( 5.1 ). Because of the risk of hepatotoxicity, JUXTAPID is available only through a restricted program called the JUXTAPID REMS Program ( 5.2 ). Prescribe JUXTAPID only to patients with a clinical or laboratory diagnosis consistent with homozygous familial hypercholesterolemia (HoFH). The safety and effectiveness of JUXTAPID have not been established in patients with hypercholesterolemia who do not have HoFH ( 1 ).

Common side effects

• Diarrhea
• Nausea
• Vomiting
• Abdominal pain
• Dyspepsia
• Weight loss
• Abdominal discomfort
• Abdominal distension
• Constipation
• Flatulence
• Increased ALT
• Chest pain

Drug interactions

• P-glycoprotein Substrates
• amprenavir
• aprepitant
• atazanavir
• boceprevir
• ciprofloxacin
• clarithromycin
• cobicistat
• conivaptan
• darunavir
• diltiazem
• erythromycin

Key clinical trials

• Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
• Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) (PHASE3)
• LOWER: Lomitapide Observational Worldwide Evaluation Registry
• Global Lomitapide Pregnancy Exposure Registry
• Study to Determine the Intra-subject Variability of Pharmacokinetics of Lomitapide in Healthy Subjects (PHASE1)
• Evaluate the Effect of Atorvastatin on the Pharmacokinetics of Lomitapide in Healthy Subjects. (PHASE1)
• Evaluate the Effect of Ethinyl Estradiol/Norgestimate on the Pharmacokinetics of Lomitapide in Healthy Female Subjects (PHASE1)
• Phase I Study of the Safety, Tolerability, PK & PD of Lomitapide in Japanese and Caucasian Subjects With Elevated LDL-C (PHASE1)

Patents

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Juxtapid CI brief — competitive landscape report
• Juxtapid updates RSS · CI watch RSS
• Chiesi portfolio CI