Lomitapide

Pronunciation: loh-MIT-a-pide.

Generic name
lomitapide
Drug class
Microsomal triglyceride transfer protein (MTP) inhibitor
Form
Capsule (5 mg, 10 mg, 20 mg; higher strengths reached by combining capsules per prescriber instructions)
Route
Oral
Legal status
Prescription-only, specialist-initiated, restricted distribution program (REMS in the United States)

Availability and supply rules vary by country

Trade names by country: United States · Canada

Australia: · Full directory

What lomitapide is and how it works

Lomitapide is a prescription medicine that blocks a protein (microsomal triglyceride transfer protein, or MTP) needed to build certain fat-carrying particles in the liver and gut. This reduces the amount of LDL cholesterol ("bad" cholesterol) the body makes and releases into the blood. It is used only for a rare, inherited form of very high cholesterol called homozygous familial hypercholesterolaemia (HoFH). (1) (2)

Because of a genuine risk of serious liver injury, lomitapide is only prescribed and dispensed under close specialist supervision, with regular liver blood tests, and (in the United States) only through a restricted distribution program. Never start, stop, or change your dose without your specialist's explicit direction.

Mechanism of action: how lomitapide works

Lomitapide directly binds and inhibits microsomal triglyceride transfer protein (MTP) in the liver and intestine, reducing the assembly and secretion of apolipoprotein-B-containing lipoproteins (VLDL in the liver, chylomicrons in the intestine), which lowers LDL-C independent of LDL-receptor function -- relevant because HoFH patients have little or no functional LDL receptor activity. (1) (2)

What lomitapide is used for

Homozygous familial hypercholesterolaemia (HoFH)

Used as an add-on to a low-fat diet and other cholesterol-lowering treatments (with or without LDL apheresis, a blood-filtering treatment) to reduce LDL cholesterol, total cholesterol, apolipoprotein B, and non-HDL cholesterol in adults (and, in the United States, children aged 2 years and older) with HoFH, a rare inherited condition causing very high cholesterol from birth. (1) (2)

Before taking lomitapide

Do not take / not appropriate for

  • Do not take lomitapide if you are pregnant. It can cause fetal harm and is contraindicated in pregnancy. (all countries) (1) (2)
  • Do not take lomitapide together with moderate or strong CYP3A4-inhibitor medicines (a class that includes many antifungal and antibiotic medicines, some HIV medicines, and others) -- these substantially raise lomitapide levels and liver-injury risk. (all countries) (1) (2)
  • Do not take lomitapide if you have moderate or severe liver impairment, active liver disease, or unexplained persistently abnormal liver blood tests. (all countries) (1) (2)
  • Do not take lomitapide if you have significant or chronic bowel disease, such as inflammatory bowel disease or a malabsorption condition. (United Kingdom, European Union) (2)
  • Do not combine lomitapide with more than 40 mg of simvastatin daily, and avoid grapefruit juice. (all countries) (2) (1)

Tell your clinician about

  • Any history of liver problems or abnormal liver blood tests.
  • Any history of bowel disease, malabsorption, or significant gastrointestinal problems.
  • All other medicines and supplements, especially anything that affects liver enzymes (CYP3A4), statins, warfarin, or bile-acid-binding medicines.
  • Whether you are pregnant, planning pregnancy, or breastfeeding -- effective contraception is required during treatment and for 2 weeks after your last dose if you can become pregnant.
  • Whether you have kidney disease requiring dialysis.
(1) (2)

How to take lomitapide

  • Taken once daily, with a full glass of water, without food -- at least 2 hours after your evening meal.
  • Your specialist will start you on a low dose and increase it gradually over weeks, based on your liver blood tests and how you tolerate the medicine.
  • Take a daily multivitamin supplement with vitamin E and essential fatty acids as directed by your specialist, since lomitapide can reduce absorption of these nutrients.
  • Do not change your dose or stop taking lomitapide without your specialist's guidance -- dose changes are tied to your liver test results.
(1) (2)

Procedures and treatment changes

Tell the treating team before surgery, procedures, or any medicine change. Do not alter treatment without the responsible clinician. (1) (2) (3) (4)

Missed dose

Contact your specialist or care team for guidance if you miss a dose. Do not double up or adjust your next dose on your own.

Overdose

Seek urgent medical attention if you think you have taken too much lomitapide, or if you develop severe stomach pain, yellowing of the skin or eyes, or other new symptoms. (1)

Side effects

Side effects and their urgency vary; report severe, persistent, or concerning symptoms.

Diarrhoea (reported in most patients in clinical trials).

What to notice: Diarrhoea (reported in most patients in clinical trials).

What to do: Discuss persistent or troublesome symptoms with the treating clinician or pharmacist. (2)

Nausea and vomiting.

What to notice: Nausea and vomiting.

What to do: Discuss persistent or troublesome symptoms with the treating clinician or pharmacist. (2) (1)

Indigestion (dyspepsia) and abdominal pain.

What to notice: Indigestion (dyspepsia) and abdominal pain.

What to do: Discuss persistent or troublesome symptoms with the treating clinician or pharmacist. (1) (2)

Decreased appetite and fatigue.

What to notice: Decreased appetite and fatigue.

What to do: Discuss persistent or troublesome symptoms with the treating clinician or pharmacist. (2)

Elevated liver enzymes on blood tests.

What to notice: Elevated liver enzymes on blood tests.

What to do: Discuss persistent or troublesome symptoms with the treating clinician or pharmacist. (1) (2)

Serious warnings

Signs of serious liver injury — boxed_warning

Symptoms: Unusual tiredness, loss of appetite, discomfort in the upper right side of the abdomen, dark urine, or yellowing of the skin or eyes (jaundice).

Action: Contact your clinician promptly and have liver blood tests checked -- lomitapide can cause serious liver injury, including a build-up of liver fat that may progress to more severe liver disease. Do not restart or change your dose after such signs without your clinician's explicit direction. (1) (2)

Signs of severe or persistent gastrointestinal symptoms — high caution

Symptoms: Severe or persistent diarrhoea, vomiting, or abdominal pain that does not settle.

Action: Contact your clinician -- gastrointestinal side effects are very common with lomitapide and may need dose adjustment or supportive treatment. (2)

Pregnancy or planning pregnancy — emergency

Symptoms: Becoming pregnant, or a missed period while taking lomitapide.

Action: Contact your clinician immediately. Lomitapide is contraindicated in pregnancy and can cause fetal harm; effective contraception is required during treatment and for 2 weeks after the last dose for anyone who can become pregnant. (1)

Signs of a serious allergic reaction — emergency

Symptoms: Swelling of the face, lips, tongue, or throat; difficulty breathing; widespread rash or hives.

Action: Seek emergency medical help immediately. (2)

Interactions

  • Moderate or strong CYP3A4-inhibitor medicines (many antifungals, macrolide antibiotics, some HIV medicines, and others) are contraindicated with lomitapide because they substantially raise lomitapide levels and liver-injury risk. (1) (2)
  • Weak CYP3A4 inhibitors require your specialist to reduce your lomitapide dose. (1)
  • Grapefruit and grapefruit juice increase lomitapide levels and should be avoided. (2)
  • Simvastatin above 40 mg daily is not recommended with lomitapide because of an increased risk of muscle problems (myopathy). (2) (1)
  • Warfarin levels (INR) can increase with lomitapide -- your clinician will monitor your INR more closely. (1) (2)
  • Bile-acid-binding medicines should be taken at least 4 hours apart from lomitapide, as they can reduce its absorption. (1)

Pregnancy, breastfeeding, kidney/liver function, age

Pregnancy

Contraindicated. Lomitapide can cause fetal harm based on animal studies. Effective contraception is required during treatment and for 2 weeks after the last dose for anyone who can become pregnant. (1) (2) (3) (4)

Breastfeeding

Not recommended -- it is not known whether lomitapide passes into breast milk; discuss the risks and benefits with your clinician. (1) (2) (3) (4)

Children

The US label extends the indication to children aged 2 years and older with HoFH; use in children elsewhere should follow the locally approved product information and specialist guidance. (1) (2) (3) (4)

Elderly

No specific dose adjustment based on age alone is described in the sources reviewed, but liver monitoring and interaction risks apply equally. (1) (2) (3) (4)

Hepatic_impairment

Contraindicated in moderate or severe hepatic impairment or active liver disease; a reduced maximum dose applies in mild hepatic impairment. (1) (2) (3) (4)

Renal_impairment

A reduced maximum dose applies in end-stage renal disease requiring dialysis. (1) (2) (3) (4)

Source_ids

US-DAILYMED-JUXTAPID-2026,UK-EMC-LOJUXTA-2026 (1) (2) (3) (4)

Monitoring

Before starting

  • Baseline liver function tests (ALT, AST, alkaline phosphatase, total bilirubin)
  • Pregnancy test and contraception plan where relevant
  • Review of other medicines for CYP3A4 interactions and statin use

During treatment

  • Liver function tests before each dose increase, and at least monthly during the first year of treatment, then quarterly, per the current product information.
  • Ongoing assessment for signs of liver injury (fatigue, appetite loss, right-upper-quadrant discomfort, dark urine, jaundice).
  • Assessment of gastrointestinal tolerability and fat-soluble vitamin status.
(1) (2)

Storage

Store capsules in their original packaging at room temperature, out of reach of children, following the exact instructions on your product's label.

Professional information

Clinician-facing context only, sourced from the current US FDA Juxtapid prescribing information and the UK/EU Lojuxta SmPC/EPAR; verify against the current full label before prescribing.

United States / United Kingdom / European Union / Canada

Indication: Homozygous familial hypercholesterolaemia (HoFH)

Dose summary: Start 5 mg once daily; titrate no sooner than 2 weeks to 10 mg, then at intervals of at least 4 weeks to 20 mg, 40 mg, to a maximum of 60 mg once daily, guided by hepatic tolerability.

Renal context: Maximum 40 mg daily in end-stage renal disease on dialysis (US label); no specific EU/UK renal dose adjustment described in the sources reviewed. (1) (2)

Mechanism of action

Hepatic guidance

Contraindicated in moderate/severe hepatic impairment or active liver disease; hepatotoxicity (transaminase elevation and hepatic steatosis) is the boxed-warning-level risk requiring scheduled liver function monitoring and dose adjustment or discontinuation per the current label's thresholds.

Overdose note

No specific antidote; manage with supportive care and close monitoring of hepatic and gastrointestinal status. (1) (2)

Frequently asked questions

Is lomitapide available in the US, UK, Europe, and Canada?

Yes. It is authorised in all of these regions -- sold as Juxtapid in the United States and Canada, and as Lojuxta in the United Kingdom and European Union -- for adults (and, in the US, children aged 2 and older) with the rare condition homozygous familial hypercholesterolaemia (HoFH). Its availability in Australia and New Zealand could not be confirmed from any live regulator source at the time of writing. (1) (2) (3) (4)

Why do I need regular liver blood tests on lomitapide?

Lomitapide carries a boxed warning for liver injury -- it can raise liver enzyme levels and increase fat stored in the liver, which may be a risk factor for more serious liver disease. Regular liver blood tests, especially before each dose increase, let your specialist catch problems early so they can adjust your treatment as needed. (1) (2)

Why is lomitapide only available through a restricted program in the US?

Because of its genuine risk of serious liver injury, the FDA requires lomitapide to be prescribed and dispensed only through the JUXTAPID REMS Program, which certifies prescribers and pharmacies and ensures patients are properly monitored. (1)

What is the mechanism of action of lomitapide?

Lomitapide directly binds and inhibits microsomal triglyceride transfer protein (MTP) in the liver and intestine, reducing the assembly and secretion of apolipoprotein-B-containing lipoproteins (VLDL in the liver, chylomicrons in the intestine), which lowers LDL-C independent of LDL-receptor function -- relevant because HoFH patients have little or no functional LDL receptor activity. (1) (2)

What is the generic name of Lomitapide?

The generic name is lomitapide. (1) (2)

What class of drug is lomitapide?

Lomitapide is classed as: Microsomal triglyceride transfer protein (MTP) inhibitor. (1) (2)

References

  1. JUXTAPID (lomitapide) capsules, for oral use -- full prescribing informationU.S. Food and Drug Administration / DailyMed (NIH), full prescribing information, Aegerion Pharmaceuticals, Inc. / Amryt Pharmaceuticals · United States · accessed 2026-07-30 · source 1
  2. Lojuxta 5 mg / 10 mg / 20 mg hard capsules -- Summary of Product Characteristicselectronic medicines compendium (emc), United Kingdom -- Chiesi/Amryt marketing authorisation holder · United Kingdom · accessed 2026-07-30 · source 2
  3. Lojuxta (lomitapide) -- EPAR medicine overviewEuropean Medicines Agency (EMA) -- EPAR for Lojuxta · European Union · accessed 2026-07-30 · source 3
  4. JUXTAPID (lomitapide) Product MonographHealth Canada product monograph, distributed via Medison Pharma / Amryt · Canada · accessed 2026-07-30 · source 4

Brand names by country

Single-ingredient lomitapide names are shown separately by country. Product availability, strengths, and supply rules can change; combination products are not included here.

Priority countries

United States

  • Juxtapid5 mg, 10 mg, 2 mg, 20 mg, 30 mg · chiesi usa, inc.

Canada

  • JUXTAPID
View brands in additional countries.

This page provides general reference information about lomitapide and is not a substitute for advice from the specialist managing your treatment. All dosing and treatment decisions should be made with your prescriber, under the restricted-access program and monitoring schedule appropriate to your country.

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