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Understanding Homozygous Familial Hypercholesterolemia (HoFH)

HoFH is a rare and life-threatening genetic disorder that causes extremely high levels of cholesterol in the blood1

Untreated HoFH may result in LDL-C levels between 400-1000 mg/dL.2,3
HoFH affects about 1:250,000 to 1:360,000 people4
HoFH is often not well controlled on lipid-lowering therapies or apheresis alone.2,5,6
ESC/EAS guidelines strongly recommend that lipid-lowering therapy be started as early as possible to help delay the onset of CVD.1,2,7
EAS, European Atherosclerosis Society; ESC, European Society of Cardiology; CVD, cardiovascular disease.

By themselves, LDL-R–regulated therapies will have reduced or limited effectiveness in HoFH

Patients with HoFH inherit a genetic mutation from each parent that causes low-density lipoprotein receptor (LDL-R) impairment, limiting the body’s ability to clear LDL-C from circulation.3

LDL-C levels may remain elevated in patients with HoFH despite treatment with traditional lipid-lowering medications because these medications exert their effects by upregulating LDL receptors, which may be absent or dysfunctional in patients with HoFH.6

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LDL-R
Negative1
Complete absence of the
LDL-R protein in receptor negative (null) patients results in <2% functionality of the pathway.
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LDL-R
Defective1
The presence of some defective LDL-R protein in receptor-defective patients results in 2%-25% functionality of the pathway.
LDL-R–negative patients have higher LDL-C levels and poorer clinical prognosis than LDL-R–defective patients.1

HoFH is difficult to treat. Most patients will not reach LDL-C threshold with standard-of-care therapies alone

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Despite maximum standard lipid-lowering therapies and following a low-fat diet, most patients with HoFH cannot get LDL-C below guideline-recommended thresholds1
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International guidelines recommend that LDL cholesterol should be at or below 70 mg/dL, and at or below 55 mg/dL for patients with signs of cardiovascular disease2,8,9
References:
1. Cuchel M, Bruckert E, Ginsberg HN, et al. Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management: a position paper from the Consensus Panel on Familial Hypercholesterolaemia of the European Atherosclerosis Society. Eur Heart J. 2014;35(32):2146-2157. doi:10.1093/eurheartj/ehu274 2. Cuchel M, Raal FJ, Hegele RA, et al. 2023 update on European Atherosclerosis Society Consensus Statement on Homozygous Familial Hypercholesterolaemia: new treatments and clinical guidance. Eur Heart J. 2023;44(25):2277-2291. doi:10.1093/ eurheartj/ehad197 3. Raal FJ, Sjouke B, Hovingh GK, Isaac BF. Phenotype diversity among patients with homozygous familial hypercholesterolemia: a cohort study. Atherosclerosis. 2016;248:238-244. doi:10.1016/j. atherosclerosis.2016.03.009 4. Fularski P, Hajdys J, Majchrowicz G, et al. Unveiling familial hypercholesterolemia—review, cardiovascular complications, lipid-lowering treatment and its efficacy. Int J Mol Sci. 2024;25(3):1637. doi:10.3390/ijms25031637 5. 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 6. Goldstein JL, Hobbs HH, Brown MS. Familial Hypercholesterolemia. In: Valle DL, Antonarakis S, Ballabio A, Beaudet AL, Mitchell GA. eds. The Online Metabolic and Molecular Bases of Inherited Disease. McGraw-Hill Education; 2019. Accessed August 25, 2025. https://ommbid.mhmedical.com/content.aspx?bookid=2709&sectionid=225539965 7. European Association for Cardiovascular Prevention & Rehabilitation, Reiner Z, Catapano AL, et al. ESC/EAS guidelines for the management of dyslipidaemias: the task force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS). Eur Heart J. 2011;32(14):1769-1818. doi:10.1093/eurheartj/ehr158 8. Mach FM, Baigent C, ESC Scientific Document Group et al. 2019 ESC/EAS Guidelines for the management of dyslipidaemias: lipid modification to reduce cardiovascular risk: The Task Force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and European Atherosclerosis Society (EAS). Eur Heart J. 2020; 41:111–188, https://doi.org/10.1093/eurheartj/ehz455. 9. Grundy SM, Stone NJ, Bailey AL, et al. 2018 AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA guideline on the management of blood cholesterol: a report of the American College of Cardiology/American Heart Association Task Force on Clinical Practice Guidelines. Circulation. 2019;139:e1082–e1143.

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:

  • 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.