Lomitapide

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.

• JUXTAPID is indicated as an adjunct to a low-fat diet and exercise and other low density lipoprotein cholesterol (LDL-C) therapies to reduce LDL-C in adults and pediatric patients aged 2 years and older with homozygous familial hypercholesterolemia (HoFH). JUXTAPID is a microsomal triglyceride transfer protein inhibitor indicated as an adjunct to a low-fat diet and exercise and other low-density lipoprotein cholesterol (LDL-C) therapies, to reduce LDL-C in adult and pediatric patients aged 2 years and older with HoFH.ICD-10: E78.00

• JUXTAPID is contraindicated in the following conditions: Pregnancy [see Warnings and Precautions (5.3) and Use in Specific Populations (8.1) ] . Concomitant administration of JUXTAPID with moderate or strong CYP3A4 inhibitors, as this can increase JUXTAPID exposure [see Warnings and Precautions (5.6) , Drug Interactions (7.1) , and Clinical Pharmacology (12.3) ].contraindicated

Before treatment, measure ALT, AST, alkaline phosphatase, and total bilirubin; obtain a negative pregnancy test in females of reproductive potential; initiate a low-fat diet supplying <20% of energy from fat or less than 30 grams of fat, whichever is less. ( 2.1 ). The recommended initiation dosage is ( 2.2 ): 2 mg for patients aged 2 to 15 years. 5 mg for patients aged 16 years and older. Follow the titration schedule presented in Table 1 according to the patient's age. Select the dosage based on the recommended target LDL-C, safety, and tolerability ( 2.2 ). For pediatric patients, if a patient crosses over into the next age category, escalate the dose of JUXTAPID up to the maximum recommended dose applicable for the new age group ( 2.2 ). Measure transaminases prior to any dosage increase. If transaminases are abnormal, reduce or withhold dosing of JUXTAPID and monitor as recommended ( 5.1 ). Table 1: Recommended JUXTAPID Dosage and Titration Schedule Age group (years) JUXTAPID Dose 2 mg 5 mg 10 mg 20 mg 40 mg 60 mg W:Weeks; ---: Not a recommended dosage; 1 Maximum recommended dosage.

Embryo-Fetal Toxicity: May cause fetal harm. Advise females of reproductive potential of the potential risk to the fetus and to use effective contraception. Discontinue JUXTAPID if pregnancy detected ( 5.3 ). Gastrointestinal adverse reactions occur in 93% of adult and 72% of pediatric patients and could affect absorption of concomitant oral medications ( 5.5 ). 5.1 Risk of Hepatotoxicity JUXTAPID can cause elevations in transaminases and hepatic steatosis in adults and pediatric patients, as described below [see Warnings and Precautions (5.2) ] . JUXTAPID may induce steatohepatitis, which can progress to cirrhosis over several years. Clinical trials of JUXTAPID for HoFH would have been unlikely to detect this adverse outcome given their size and duration [see Clinical Studies (14) ] . Elevation of Transaminases Elevations in transaminases (ALT and/or AST) are associated with JUXTAPID. In the 78-week adult clinical trial, 10 (34%) of the 29 patients with HoFH had at least one elevation in ALT or AST ≥3 times ULN, and 4 (14%) of the patients had at least one elevation in ALT or AST ≥5 times ULN. There were no concomitant or subsequent clinically meaningful elevations in bilirubin, INR, or alkaline phosphatase [see Adverse Reactions (6.1) ]. No patients discontinued prematurely because of elevated transaminases.

The following important adverse reactions have been observed and are discussed in detail in other sections of the label: Risk of hepatotoxicity [see Warnings and Precautions (5.1) ] Reduced absorption of fat-soluble vitamins, and serum fatty acids [see Warnings and Precautions (5.4) ] Gastrointestinal adverse reactions [see Warnings and Precautions (5.5) ] Most common adverse reactions in adult patients (incidence ≥10%) are diarrhea, nausea, dyspepsia, vomiting, and abdominal pain (6.1). Most common adverse reactions in pediatric patients aged 5 to 17 years old (incidence ≥15%) are abdominal pain, alanine aminotransferase increased, aspartate aminotransferase increased, diarrhea, and vomiting ( 6.1 ). To report SUSPECTED ADVERSE REACTIONS, contact Chiesi Farmaceutici S.p.A. at 1-888-661-9260 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Adults with HoFH One single-arm, open-label, 78-week trial has been conducted in 29 adult patients with HoFH, 23 of whom completed at least one year of treatment. The initial dosage of JUXTAPID was 5 mg daily, with titration up to 60 mg daily during an 18-week period based on safety and tolerability.

Lactation: Breastfeeding not recommended ( 8.2 ). 8.1 Pregnancy Pregnancy Exposure There is a registry that monitors pregnancy outcomes in women exposed to JUXTAPID during pregnancy. For additional information visit www.JUXTAPID.com or call the Lomitapide Observational Worldwide Exposure Registry (LOWER) at 1-877-902-4099. Healthcare professionals are encouraged to call the LOWER at 1-877-902-4099 to enroll patients who become pregnant during JUXTAPID treatment. Risk Summary Based on findings from animal studies, JUXTAPID use is contraindicated in pregnancy since it may cause fetal harm [see Contraindications (4) , Warnings and Precautions (5.3) ]. Available human data are insufficient to draw conclusions about any drug-associated risks for major birth defects, miscarriage, or adverse maternal or fetal outcomes. However, in animal reproduction studies, lomitapide was teratogenic in rats at clinically relevant exposures and in ferrets at exposures estimated to be less than human therapeutic exposure at 60 mg when administered during organogenesis, based on AUC comparisons. Embryo-fetal lethality was observed in rabbits at 6-times the maximum recommended human dose (MRHD) of 60 mg based on body surface area. If pregnancy is detected, discontinue JUXTAPID. The estimated background risk of major birth defects and miscarriage for the indicated population is unknown. In the U.S.

Metabolism

Lomitapide pharmacokinetics is approximately dose-proportional for oral single doses from 10 to 100 mg. Following the maximum recommended dose of 60 mg daily in patients ≥18 years old, the steady state exposure (i.e., AUC tau with tau=24 hours) and C max were predicted to be 116.34 ng/mL∙h (with 90% prediction interval of 41.7 to 362.6 ng/mL∙h) and 5.74 ng/mL (2.00 to 17.9 ng/mL) respectively (see Table 11 ).

There is no specific treatment in the event of overdose of JUXTAPID. In the event of overdose, the patient should be treated symptomatically, and supportive measures instituted as required. Liver-related tests should be monitored. Hemodialysis is unlikely to be beneficial given that lomitapide is highly protein-bound.

• Dec 21, 2012NDANDA203858Chiesi

1. 1Diarrhoea1,06628%
2. 2Weight Decreased84222%
3. 3Therapy Cessation79221%
4. 4Nausea55115%
5. 5Abdominal Pain Upper2817.5%
6. 6Flatulence2416.4%
7. 7Abdominal Discomfort2396.4%
8. 8Vomiting2165.8%
9. 9Fatigue2065.5%
10. 10Off Label Use2065.5%
11. 11Hepatic Enzyme Increased1885.0%
12. 12Drug Dose Omission1844.9%
13. 13Inappropriate Schedule Of Drug Administration1844.9%
14. 14Constipation1684.5%
15. 15Abdominal Pain1654.4%

The 10 most recently updated of 20 ClinicalTrials.gov registrations naming Lomitapide as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• LOWER: Lomitapide Observational Worldwide Evaluation Registry
  Completed · Observational · 260 enrolled · Amryt Pharma
  NCT02135705updated 2026-05-12
• Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
  Active not recruiting · Observational · 73 enrolled · Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)
  NCT06832371updated 2026-02-20
• Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
  Completed · Phase 3 · Interventional · 46 enrolled · Amryt Pharma
  NCT04681170updated 2025-08-27
• Global Lomitapide Pregnancy Exposure Registry
  Terminated · Observational · 5 enrolled · Amryt Pharma
  NCT02399839updated 2022-12-16
• Study to Determine the Intra-subject Variability of Pharmacokinetics of Lomitapide in Healthy Subjects
  Completed · Phase 1 · Interventional · 15 enrolled · Aegerion Pharmaceuticals, Inc.
  NCT01915771updated 2020-03-11
• Evaluate the Effect of Atorvastatin on the Pharmacokinetics of Lomitapide in Healthy Subjects.
  Completed · Phase 1 · Interventional · 32 enrolled · Aegerion Pharmaceuticals, Inc.
  NCT02080455updated 2020-03-10
• Evaluate the Effect of Ethinyl Estradiol/Norgestimate on the Pharmacokinetics of Lomitapide in Healthy Female Subjects
  Completed · Phase 1 · Interventional · 32 enrolled · Aegerion Pharmaceuticals, Inc.
  NCT02080468updated 2019-03-11
• Phase I Study of the Safety, Tolerability, PK & PD of Lomitapide in Japanese and Caucasian Subjects With Elevated LDL-C
  Completed · Phase 1 · Interventional · 72 enrolled · Aegerion Pharmaceuticals, Inc.
  NCT01760187updated 2018-11-20
• Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy
  Completed · Phase 3 · Interventional · 9 enrolled · Aegerion Pharmaceuticals, Inc.
  NCT02173158updated 2018-10-10
• Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia
  Completed · Phase 3 · Interventional · 19 enrolled · Aegerion Pharmaceuticals, Inc.
  NCT00943306updated 2018-06-13

Frequently asked questions

How does Lomitapide work?

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.

What is Lomitapide used for?

According to FDA labeling, Lomitapide carries indications including: JUXTAPID is indicated as an adjunct to a low-fat diet and exercise and other low density lipoprotein cholesterol (LDL-C) therapies to reduce LDL-C in adults and pediatric patients aged 2 years and older with homozygous familial hypercholesterolemia (HoFH). JUXTAPID is a microsomal triglyceride transfer protein inhibitor indicated as an adjunct to a low-fat diet and exercise and other low-density lipoprotein cholesterol (LDL-C) therapies, to reduce LDL-C in adult and pediatric patients aged 2 years and older with HoFH.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Lomitapide?

Lomitapide is classified as Other lipid modifying agents, Microsomal Triglyceride Transfer Protein Inhibitor, Cytochrome P450 3A4 Inhibitors, Microsomal Triglyceride Transfer Protein Inhibitors, P-Glycoprotein Inhibitors, Decreased Cholesterol Synthesis.

What are the brand names for Lomitapide?

Lomitapide is marketed under brand names including Juxtapid.

What are the contraindications for Lomitapide?

Lomitapide labeling lists contraindications including: JUXTAPID is contraindicated in the following conditions: Pregnancy [see Warnings and Precautions (5.3) and Use in Specific Populations (8.1) ] . Concomitant administration of JUXTAPID with moderate or strong CYP3A4 inhibitors, as this can increase JUXTAPID exposure [see Warnings and Precautions (5.6) , Drug Interactions (7.1) , and Clinical Pharmacology (12.3) ].. Always consult the full prescribing information and a clinician.