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Efficacy of Juxtapid

Juxtapid offers long-term efficacy supported by clinical evidence

Juxtapid was evaluated in a 78-week, open-label, single-arm phase 3 clinical trial.1,2

Twenty-nine HoFH patients were treated with Juxtapid and standard-of-care treatment, including statins, ezetimibe, and lipoprotein apheresis (in 93%, 76%, and 62% of patients, respectively).1,2

Timeline of Juxtapid clinical studies showing a 6-week run-in period followed by a core study of 78 weeks and a long-term extension study of 216 weeks. Key points include treatment start at week 0, primary endpoint at week 26, long-term safety endpoint at week 78, and primary endpoint at week 126. Timeline of Juxtapid clinical studies showing a 6-week run-in period followed by a core study of 78 weeks and a long-term extension study of 216 weeks. Key points include treatment start at week 0, primary endpoint at week 26, long-term safety endpoint at week 78, and primary endpoint at week 126.
logo-sign The primary study endpoint was the percentage reduction in LDL-C at week 26; patients then entered the 52-week safety phase1,2
logo-sign After 78 weeks, eligible patients were invited to enter a long-term extension, which lasted up to 4.2 years4

Juxtapid helps patients reduce their LDL-C levels and maintain these levels over time

Durable response—patients maintained LDL-C reductions through the 78-week core study and the 216-week long-term extension.1,2,4

This study was a 78-week, single-arm, open-label, multicenter, international study of 29 adults (age 18-55 years) with HoFH that consisted of 3 phases: a 6-week run-in period, a 26-week efficacy phase, and a 52-week safety phase. Concomitant lipid-lowering therapies at baseline included one or more of the following: statins (93%), ezetimibe (76%), nicotinic acid (10%), bile acid sequestrant (3%), and fibrate (3%); 18 (62%) were receiving apheresis. Concomitant lipid-lowering therapies including apheresis were to remain unchanged from baseline to week 26.1
Mean percentage change in LDL-C from baseline1,2,4
LDL-C reductions with Juxtapid: 40% in intent-to-treat population at week 26, 50% in patients completing treatment per protocol at week 26, and 45.5% in patients enrolled in the long-term extension at week 126. LDL-C reductions with Juxtapid: 40% in intent-to-treat population at week 26, 50% in patients completing treatment per protocol at week 26, and 45.5% in patients enrolled in the long-term extension at week 126.
logo-sign
All patients started at a daily dosage of 5 mg that was titrated to a daily dosage of 10 mg, 20 mg, 40 mg, and 60 mg based on individual tolerability and transaminase levels.2

Juxtapid significantly reduced additional key lipids often elevated in patients with HoFH1

Similar efficacy results were reported for LDL-C, TC, apo B, and non–HDL-C, and were sustained over 78 weeks.2

Apo B, apolipoprotein B; HDL-C, high-density lipoprotein cholesterol; non–HDL-C, non–high-density lipoprotein cholesterol; TC, total cholesterol; TG, triglyceride; VLDL-C, very low-density lipoprotein cholesterol.

Percentage change from baseline in lipids and lipoproteins at 26 weeks (N=29)
/_assets/images/v3/lipids/lipids-chart-mobile.svg/_assets/images/v3/lipids/lipids-chart-mobile.svg
aStatistically significant compared with baseline based on the prespecified gatekeeping method of controlling Type 1 error among the key secondary end points.
bMedian value with interquartile range and median percentage change shown for TGs.

LDL-C reductions maintained for more than 5 years4

Mean percentage change in LDL-C vs baseline4

Juxtapid works to achieve target LDL-C levels

Most patients in the clinical trial reached target LDL-C levels at any time through week 2464:

74%
Patients who reached
LDL-C target of <100 mg/dL
(n=14)
58%
Patients who reached
LDL-C target of <70 mg/dL
(n=11)

Juxtapid works for patients,
long-term4

74-percent-chart

In the study extension

Most patients remained on Juxtapid for almost 5 years (246 weeks).

References:
1. JUXTAPID (lomitapide) Prescribing Information. Chiesi Farmaceutici S.p.A.; 2025. 2. Cuchel M, Meagher EA, du Toit Theron H, et al. Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. Lancet. 2013;381(9860):40-46. doi:10.1016/S0140-6736(12)61731-0 3. Cuchel M, Blom DJ, Averna MR. Clinical experience of lomitapide therapy in patients with homozygous familial hypercholesterolaemia. Atheroscler Suppl. 2014;15(2):33-45. doi:10.1016/j.atherosclerosissup.2014.07.005 4. Blom DJ, Averna MR, Meagher EA, et al. Long-term efficacy and safety of the microsomal triglyceride transfer protein inhibitor lomitapide in patients with homozygous familial hypercholesterolemia. Circulation. 2017;136(3):332-335. doi:10.1161/CIRCULATIONAHA.117.028208

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION:

Juxtapid (lomitapide) capsules 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).

IMPORTANT SAFETY INFORMATION:

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.

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.

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.

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. 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.

CONTRAINDICATIONS:

  • Pregnancy
  • Concomitant administration with moderate or strong CYP3A4 inhibitors
  • Moderate or severe hepatic impairment and active liver disease, including unexplained persistent elevations of serum transaminases

WARNINGS AND PRECAUTIONS:

Juxtapid can cause elevations in transaminases and hepatic steatosis in adult and pediatric patients. Juxtapid may induce steatohepatitis, which can progress to cirrhosis over several years.  Modify the dose of Juxtapid if elevations of transaminases are observed and discontinue Juxtapid for persistent or clinically significant elevations. If transaminase elevations are accompanied by clinical symptoms of liver injury, such as nausea, vomiting, abdominal pain, fever, jaundice, lethargy, flu-like-symptoms, increases in bilirubin ≥2x ULN, or active liver disease, discontinue treatment with Juxtapid and identify the probable cause. Use Juxtapid with caution when co-administered with agents known to be hepatotoxic. Alcohol may increase levels of hepatic fat and induce or exacerbate liver injury.  Drinking more than one alcoholic drink per day is not recommended for patients taking Juxtapid.

Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment. During the first year, measure liver-related tests (ALT and AST, at a minimum) prior to each increase in dose or monthly, whichever occurs first. After the first year, do these tests at least every 3 months and before any increase in dose.

Juxtapid may cause fetal harm when administered to a pregnant woman. Females of reproductive potential should have a negative pregnancy test before starting Juxtapid and should use effective contraception during therapy with Juxtapid and for two weeks after the final dose.  If pregnancy is detected, discontinue Juxtapid.

Given its mechanism of action in the small intestine, Juxtapid may reduce the absorption of fat-soluble nutrients. Patients ages 9 years and older treated with Juxtapid should take daily supplements that contain 400 international units (IU) vitamin E (or 200 IU vitamin E for pediatric patients aged 2 to 8 years) and at least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA).

Gastrointestinal adverse reactions are common and may lead to treatment discontinuation. Instruct patients to stop Juxtapid and contact their healthcare provider if severe diarrhea occurs, or if they experience symptoms of volume depletion such as lightheadedness, decreased urine output, or tiredness. In such cases, consider reducing the dose or suspending use of Juxtapid. To reduce the risk of gastrointestinal adverse reactions, instruct patients or their caregiver(s) to adhere to a low-fat diet supplying <20% of energy from fat or less than 30 grams of fat, whichever is less. Increase the dosage of Juxtapid gradually. Individualize the maximum daily fat goal based on caloric needs due to age, growth, activity level, and tolerability. Monitor growth and weight loss in pediatric patients who are below the 10th percentile for height and BMI, particularly for those who just have a body weight ≥15 kg.

Weak CYP3A4 inhibitors can increase the exposure of lomitapide approximately 2-fold; therefore, when Juxtapid is administered with weak CYP3A4 inhibitors, the dose of Juxtapid should be decreased by half.  Careful titration may then be considered based on LDL-C response of safety/tolerability to half the maximum recommended dosage except when co-administered with oral contraceptives only, in which case the maximum recommended Juxtapid dose is approximately two thirds the maximum recommended dose.

Due to risk of myopathy associated with simvastatin or lovastatin, doses of these agents should be limited when co-administered with Juxtapid.

Juxtapid increases the plasma concentrations of warfarin. Increases or decreases in the dose of Juxtapid may lead to supra- or subtherapeutic anticoagulation, respectively. Patients taking warfarin should undergo regular monitoring of the INR, especially after any changes in Juxtapid dosage.

Avoid use of Juxtapid in patients with rare hereditary diseases of galactose intolerance.

ADVERSE REACTIONS:

The most common adverse reactions in adult patients (incidence ≥28%) are diarrhea, nausea, vomiting, dyspepsia, and abdominal pain.

The most common adverse reactions in pediatric patients aged 5 to 17 years old (incidence ≥15%) are diarrhea, abdominal pain, alanine aminotransferase increased, aspartate aminotransferase increased, and vomiting.

REPORTING OF ADVERSE REACTIONS:

All healthcare professionals should report all suspected adverse reactions. Please contact Chiesi Farmaceutici S.p.A. at 1-888-661-9260 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Prescribing Information including BOXED WARNING.

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION:

Juxtapid (lomitapide) capsules 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).

IMPORTANT SAFETY INFORMATION:

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.

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.

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.

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. 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.

CONTRAINDICATIONS:

WARNINGS AND PRECAUTIONS:

Juxtapid can cause elevations in transaminases and hepatic steatosis in adult and pediatric patients. Juxtapid may induce steatohepatitis, which can progress to cirrhosis over several years.  Modify the dose of Juxtapid if elevations of transaminases are observed and discontinue Juxtapid for persistent or clinically significant elevations. If transaminase elevations are accompanied by clinical symptoms of liver injury, such as nausea, vomiting, abdominal pain, fever, jaundice, lethargy, flu-like-symptoms, increases in bilirubin ≥2x ULN, or active liver disease, discontinue treatment with Juxtapid and identify the probable cause. Use Juxtapid with caution when co-administered with agents known to be hepatotoxic. Alcohol may increase levels of hepatic fat and induce or exacerbate liver injury.  Drinking more than one alcoholic drink per day is not recommended for patients taking Juxtapid.

Measure ALT, AST, alkaline phosphatase, and total bilirubin before initiating treatment. During the first year, measure liver-related tests (ALT and AST, at a minimum) prior to each increase in dose or monthly, whichever occurs first. After the first year, do these tests at least every 3 months and before any increase in dose.

Juxtapid may cause fetal harm when administered to a pregnant woman. Females of reproductive potential should have a negative pregnancy test before starting Juxtapid and should use effective contraception during therapy with Juxtapid and for two weeks after the final dose.  If pregnancy is detected, discontinue Juxtapid.

Given its mechanism of action in the small intestine, Juxtapid may reduce the absorption of fat-soluble nutrients. Patients ages 9 years and older treated with Juxtapid should take daily supplements that contain 400 international units (IU) vitamin E (or 200 IU vitamin E for pediatric patients aged 2 to 8 years) and at least 200 mg linoleic acid, 210 mg alpha-linolenic acid (ALA), 110 mg eicosapentaenoic acid (EPA), and 80 mg docosahexaenoic acid (DHA).

Gastrointestinal adverse reactions are common and may lead to treatment discontinuation. Instruct patients to stop Juxtapid and contact their healthcare provider if severe diarrhea occurs, or if they experience symptoms of volume depletion such as lightheadedness, decreased urine output, or tiredness. In such cases, consider reducing the dose or suspending use of Juxtapid. To reduce the risk of gastrointestinal adverse reactions, instruct patients or their caregiver(s) to adhere to a low-fat diet supplying <20% of energy from fat or less than 30 grams of fat, whichever is less. Increase the dosage of Juxtapid gradually. Individualize the maximum daily fat goal based on caloric needs due to age, growth, activity level, and tolerability. Monitor growth and weight loss in pediatric patients who are below the 10th percentile for height and BMI, particularly for those who just have a body weight ≥15 kg.

Weak CYP3A4 inhibitors can increase the exposure of lomitapide approximately 2-fold; therefore, when Juxtapid is administered with weak CYP3A4 inhibitors, the dose of Juxtapid should be decreased by half.  Careful titration may then be considered based on LDL-C response of safety/tolerability to half the maximum recommended dosage except when co-administered with oral contraceptives only, in which case the maximum recommended Juxtapid dose is approximately two thirds the maximum recommended dose.

Due to risk of myopathy associated with simvastatin or lovastatin, doses of these agents should be limited when co-administered with Juxtapid.

Juxtapid increases the plasma concentrations of warfarin. Increases or decreases in the dose of Juxtapid may lead to supra- or subtherapeutic anticoagulation, respectively. Patients taking warfarin should undergo regular monitoring of the INR, especially after any changes in Juxtapid dosage.

Avoid use of Juxtapid in patients with rare hereditary diseases of galactose intolerance.

ADVERSE REACTIONS:

The most common adverse reactions in adult patients (incidence ≥28%) are diarrhea, nausea, vomiting, dyspepsia, and abdominal pain.

The most common adverse reactions in pediatric patients aged 5 to 17 years old (incidence ≥15%) are diarrhea, abdominal pain, alanine aminotransferase increased, aspartate aminotransferase increased, and vomiting.

REPORTING OF ADVERSE REACTIONS:

All healthcare professionals should report all suspected adverse reactions. Please contact Chiesi Farmaceutici S.p.A. at 1-888-661-9260 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see Prescribing Information including BOXED WARNING.