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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.; 2024. 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

IMPORTANT SAFETY INFORMATION

INDICATION:

Juxtapid is a microsomal triglyceride transfer protein inhibitor indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

LIMITATIONS OF USE:

The safety and effectiveness of Juxtapid have not been established in patients with hypercholesterolemia who do not have HoFH, including those with heterozygous familial hypercholesterolemia (HeFH). The effect of Juxtapid on cardiovascular morbidity and mortality has not been determined.

WARNING: RISK OF HEPATOTOXICITY

Juxtapid can cause elevations in transaminases. In the Juxtapid clinical trial, 10 (34%) of the 29 patients treated with Juxtapid had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3x upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase.

Juxtapid also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy. 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 ≥3x 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 of moderate or strong CYP3A4 inhibitors
  • Moderate or severe hepatic impairment or active liver disease including unexplained persistent elevations of serum transaminases

WARNINGS AND PRECAUTIONS

Juxtapid can cause elevations in transaminases and hepatic steatosis. Although cases of hepatic failure have not been reported, there is concern that Juxtapid could 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.

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. The recommended maximum dosage of Juxtapid is 40 mg daily when used concomitantly with oral contraceptives.

Given its mechanism of action in the small intestine, Juxtapid may reduce the absorption of fat-soluble nutrients. Patients treated with Juxtapid should take daily supplements that contain 400 international units vitamin E 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, patients should adhere to a low-fat diet supplying less than 20% of energy from fat and the dosage of Juxtapid should be increased gradually.

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 and the recommended maximum dosage of Juxtapid is 30 mg daily. The recommended maximum dosage is 40 mg daily when used concomitantly with oral contraceptives. Strong and moderate CYP3A4 inhibitors should not be used with Juxtapid. Patients taking Juxtapid 5 mg daily may continue with the same dosage.

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 were gastrointestinal, reported by 27 (93%) of 29 patients. Adverse reactions reported by 8 (28%) or more patients in the HoFH clinical trial included diarrhea, nausea, vomiting, dyspepsia and abdominal pain. Other common adverse reactions, reported by 5 to 7 (17-24%) patients, included weight loss, abdominal discomfort, abdominal distension, constipation, flatulence, increased ALT, chest pain, influenza, nasopharyngitis, and fatigue.

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 Full Prescribing Information including BOXED WARNING for the risk of hepatotoxicity.

IMPORTANT SAFETY INFORMATION

INDICATION:

Juxtapid is a microsomal triglyceride transfer protein inhibitor indicated as an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B), and non-high-density lipoprotein cholesterol (non-HDL-C) in patients with homozygous familial hypercholesterolemia (HoFH).

LIMITATIONS OF USE:

The safety and effectiveness of Juxtapid have not been established in patients with hypercholesterolemia who do not have HoFH, including those with heterozygous familial hypercholesterolemia (HeFH). The effect of Juxtapid on cardiovascular morbidity and mortality has not been determined.

WARNING: RISK OF HEPATOTOXICITY

Juxtapid can cause elevations in transaminases. In the Juxtapid clinical trial, 10 (34%) of the 29 patients treated with Juxtapid had at least one elevation in alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥3x upper limit of normal (ULN). There were no concomitant clinically meaningful elevations of total bilirubin, international normalized ratio (INR), or alkaline phosphatase.

Juxtapid also increases hepatic fat, with or without concomitant increases in transaminases. The median absolute increase in hepatic fat was 6% after both 26 and 78 weeks of treatment, from 1% at baseline, measured by magnetic resonance spectroscopy. 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 ≥3x 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. Although cases of hepatic failure have not been reported, there is concern that Juxtapid could 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.

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. The recommended maximum dosage of Juxtapid is 40 mg daily when used concomitantly with oral contraceptives.

Given its mechanism of action in the small intestine, Juxtapid may reduce the absorption of fat-soluble nutrients. Patients treated with Juxtapid should take daily supplements that contain 400 international units vitamin E 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, patients should adhere to a low-fat diet supplying less than 20% of energy from fat and the dosage of Juxtapid should be increased gradually.

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 and the recommended maximum dosage of Juxtapid is 30 mg daily. The recommended maximum dosage is 40 mg daily when used concomitantly with oral contraceptives. Strong and moderate CYP3A4 inhibitors should not be used with Juxtapid. Patients taking Juxtapid 5 mg daily may continue with the same dosage.

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 were gastrointestinal, reported by 27 (93%) of 29 patients. Adverse reactions reported by 8 (28%) or more patients in the HoFH clinical trial included diarrhea, nausea, vomiting, dyspepsia and abdominal pain. Other common adverse reactions, reported by 5 to 7 (17-24%) patients, included weight loss, abdominal discomfort, abdominal distension, constipation, flatulence, increased ALT, chest pain, influenza, nasopharyngitis, and fatigue.

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 Full Prescribing Information including BOXED WARNING for the risk of hepatotoxicity.