Lomitapide (Juxtapid) for the Treatment of Hyperlipidemia

Lomitapide (Juxtapid)

Drug: Lomitapide

Brand Names: Juxtapid, Lojuxta

Manufacturer: Aegerion Pharmaceuticals [Ref]

Date of Approval: 2012

Primary Indication: Homozygous Familial Hypercholesterolemia

Lomitapide (Juxtapid) is an oral medicine, taken once daily, that has been approved for the treatment of patients with Homozygous Familial Hypercholesterolemia.

Homozygous Familial Hypercholesterolemia (HoFH) is an autosomal recessive disorder that manifests early in childhood.

It is a severe form of lipid disorder that is associated with early (in childhood) cardiovascular diseases such as angina and myocardial infarction.

On the other hand, Heterozygous Familial Hypercholesterolemia (HeFH) is a relatively milder form of the disease manifesting in adult life.

The table below summarizes the key differences between the homozygous and the heterozygous variants of Familial Hypercholesterolemias:




Inheritance patternAutosomal recessiveAutosomal dominant
Cholesterol levelsLDL cholesterol usually >400 mg/dlLDL cholesterol usually >190 mg/dl
Onset of symptomsSymptoms appear in childhoodSymptoms appear in adult life
Severity of symptomsSevereLess severe
TreatmentRequires aggressive treatment, such as LDL apheresis or liver transplantCan be managed with medications
PrognosisHigher risk of heart disease and other complicationsLower risk of complications

Drugs approved for the treatment of Homozygous Familial Hypercholesterolemia:

Statins don’t work here. Only a few drugs have been approved and are effective in lowering LDL cholesterol and the risk of cardiovascular diseases. These include:

A summary of the above three drugs approved for the treatment of Homozygous Familial Hypercholesterolemia is given in the table below:





Drug classAntisense oligonucleotideMicrosomal triglyceride transfer protein inhibitorPCSK9 inhibitor
Mechanism of actionReduces production of apolipoprotein BInhibits assembly and secretion of LDL cholesterolBlocks PCSK9 protein, increasing the number of LDL receptors in the liver
AdministrationSubcutaneous injection once a weekOral capsule once a daySubcutaneous injection every two to four weeks
Common side effectsInjection site reactions, flu-like symptoms, liver problemsNausea, diarrhea, vomiting, liver problemsInjection site reactions, flu-like symptoms
Approved indicationsHoFHHeFHHoFH, HeFH, and other high-risk patients with elevated LDL cholesterol levels
Use with other drugsYesYesYes
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Lomitapide (Juxtapid) for the treatment of Hyperlipidemia:

Among the available drugs for the treatment of HoFH (Homozygous Familial Hypercholesterolemia), Lomitapide is the only medicine that is available in an oral capsule formulation.

It has a different mechanism of action than statins and other lipid-lowering drugs. Lomitapide blocks the assembly of apo-B-containing lipoproteins and the secretion of LDL by inhibiting the key protein, MTP (microsomal triglyceride transfer protein).

Lomitapide blocks the production of VLDl in the liver cells and chylomicrons in the intestine. Chylomicrons are large particles containing various lipid particles including triglycerides. Blocking the assembly and transport of chylomicrons by Lomitapide can result in diarrhea.

Diarrhea is especially bothersome in patients who are taking high amounts of fats in their diet. It is essential, not only to reduce the side effects of Lomitapide but also to lower plasma lipids by reducing dietary fats to less than at least 20% of the total calorie intake per day.

Another important side effect, based on the mechanism of action of Lomitapide, is the deposition of more and more fats in the liver which can damage the liver.

A larger number of patients developed subclinical and clinical hepatitis manifesting as abnormal liver functions (LFTs).

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Efficacy of Lomitapide (Juxtapid) in HoFH:

Lomitapide has been studied in patients who were diagnosed with HoFH. The drug lowers the plasma LDL by up to 50% from the baseline.

It may reduce the need to undergo lipid apheresis which is a kind of procedure in which the lipids (LDL and apolipoprotein B – ApoB particles) in the blood are removed by the mechanism of filtration.

Study 1:

In one study conducted on 75 patients diagnosed with HoFH, Lomitapide administration significantly lowered plasma LDL levels.

The study was conducted for a median duration of 19 months. The following results were obtained:

LDL-C Reduction60% reduction from baseline
LDL-C Achieving Target Levels
  • 32% <100 mg/dL,
  • 18.7% <70 mg/dL
LDL Apheresis36.8% of patients discontinued
Adverse EventsGI side effects in 40% of patients
Hepatic SteatosisModest increase observed
Liver StiffnessRemained within the normal range in 30 patients
Cardiovascular EventsLower incidence after 2 years of treatment

Case reports:

Lomitapide is one of the options for patients with HoFH where lipid apheresis is not available. A report of two patients in Saudi Arabia who were started on Lomitapide treatment resulted in 87% and 45% reduction in the plasma LDL levels.

However, one patient had to discontinue the treatment because of the non-availability of the drug while the other patient died at the age of 26 during a cardiac surgical intervention.

Another small study:

In another small study, comprising 12 patients diagnosed with HoFH, Lomitapide resulted in an LDL reduction of about 28%, however, GI side effects were very common and the primary reason for discontinuing the drug.

The table below summarizes the findings of the study:



Number of patients12
AgeMean age: 44 ± 18 years
Age at HoFH diagnosis2 to 59 years
MedicationsAll patients on statin and ezetimibe
5 patients received LDL apheresis
LDL-C reduction with lomitapide38%


A meta-analysis of 18 studies and case reports evaluated the efficacy and safety of Lomitapide in patients with HoFH [Ref].

The results of the meta-analysis are summarized in the table below:


Sample size

Age range

Lomitapide dose

LDL-C reduction

Apo B reduction


Sperlongano et al.2Not specifiedNot specified78% (patient 1), 86% (patient 2)Not specifiedMild GI symptoms
Roeters et al.4Adultmaximum 1.0 mg/kg/d35-73%Not specifiedMild GI symptoms in 3 patients, elevated ALT levels in 1 patient
Mahzari et al.2Not specifiedNot specified55%Not specifiedNo severe AEs
Suppressa et al.128 years oldUp to 30 mg/dNot specifiedNot specifiedNo CVD complications
Cuchel et al.618-40 years old0.03-1.0 mg/kg/d50.9%55.6%Elevated ALT levels and hepatic fat accumulation
Stefanutti et al.7Adult5-60 mg/d>50% (3 patients)Not specifiedMild GI symptoms

Note: AEs = adverse events; LDL-C = low-density lipoprotein cholesterol; Apo B = apolipoprotein B; CVD = cardiovascular disease; LA = lipoprotein apheresis; LLT = lipid-lowering therapy

HOFH in Children: Results of the above meta-analysis:



Age Range

Lomitapide Dose

LDL-C Reduction

Apo B Reduction

Severe AEs

Cuchel et al.618-400.03-1.0 mg/kg/d50.9%55.6%Elevated ALT levels and hepatic fat accumulation
Stefanutti et al.7N/A5-60 mg/d>50% reduction in 3 patientsN/AN/A
Ben-omran et al.1111.6 ± 1.1 years24.5 ± 4.3 mg/d6 patients reached LDL <135 mg/dLN/AGI side effects and raised ALT
Mahzari et al.2N/AN/A87%N/AN/A
Kolovou et al.18 years2.5-40 mg/dN/AN/ANo side effects reported
Chacra et al.17.6 years20 mg/d37%N/ADiarrhea, progression of atherosclerosis
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In Summary:

Although there are availability issues and the drug is very expensive, Lomitapide is one of the key drugs that can significantly lower the LDL cholesterol levels in patients with HoFH (homozygous familial hypercholesterolemia).

Statins in combination with Ezetimibe, lipid apheresis, and PCSK-9 inhibitors are currently being used but most patients respond only partially to these treatments.

In addition, Lipid apheresis is an effective way of lowering LDL cholesterol but its effects last only for two weeks.

PCSK9 inhibitors (Evolocumab and Alirocumab) are other drugs but these are too costly, administered as injections, and not available everywhere.

Lomitapide, especially in combination with Evonocumab, can change the treatment guidelines worldwide in patients with HoFH if it is made available and the costs are lowered.

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Written by Dr. Ahmed

I am Dr. Ahmed (MBBS; FCPS Medicine), an Internist and a practicing physician. I am in the medical field for over fifteen years working in one of the busiest hospitals and writing medical posts for over 5 years.

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