Last reviewed 2026-04-20 · How we verify

LOMITAPIDE MESYLATE

LOMITAPIDE MESYLATE is a drug. It is currently FDA-approved (first approved 2012) for Reduction of LDL-C in HoFH.

Lomitapide mesylate is a marketed drug primarily indicated for the reduction of LDL-C in patients with Homozygous Familial Hypercholesterolemia (HoFH). Its key strength lies in its unique mechanism of action, which differentiates it in the treatment of this rare and severe condition. The primary risk is the expiration of the key composition patent in 2028, which could lead to increased competition from generics.

At a glance

Approved indications

• Reduction of LDL-C in HoFH

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

Serious adverse events

• Diarrhea (severe)
• Vomiting (severe)
• Increased ALT/Hepatotoxicity (severe)
• Abdominal pain/distension/discomfort (severe)
• Markedly elevated transaminases (ALT 24x ULN, AST 13x ULN)
• Transaminase elevation ≥3x ULN requiring dose reduction

Drug interactions

• Strong CYP3A4 Inhibitors
• Moderate CYP3A4 Inhibitors
• Grapefruit Juice
• Weak CYP3A4 Inhibitors
• Warfarin
• Simvastatin
• Lovastatin
• P-glycoprotein (P-gp) Substrates
• Bile Acid Sequestrants

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:

• LOMITAPIDE MESYLATE CI brief — competitive landscape report
• LOMITAPIDE MESYLATE updates RSS · CI watch RSS

Frequently asked questions about LOMITAPIDE MESYLATE

Related

• Indication: Drugs for Reduction of LDL-C in HoFH

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing