Lomitapide
Lomitapide is a microsomal triglyceride transfer protein (MTP) inhibitor used as an adjunct therapy for homozygous familial hypercholesterolemia (HoFH), a rare genetic disorder causing severely elevated LDL cholesterol. By inhibiting MTP in the liver and intestines, it prevents the formation of apo B-containing lipoproteins, significantly reducing LDL cholesterol levels in patients who have not responded to statins and other conventional therapies.
Lomitapide treats homozygous familial hypercholesterolemia by blocking lipoprotein assembly through MTP inhibition.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- C10AX12
- RxNorm
- 1364479
- Generic
- Lomitapide
- Class
- atc-c10
- Brands
- Juxtapid
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Adjunct treatment for homozygous familial hypercholesterolemia (HoFH) in combination with a low-fat diet and other lipid-lowering therapies for patients not adequately controlled with existing treatments.
Directly binds and inhibits microsomal triglyceride transfer protein (MTP) in the endoplasmic reticulum of enterocytes and hepatocytes, preventing assembly of apo B-containing lipoproteins (chylomicrons and VLDL), thereby reducing LDL cholesterol synthesis.
Requires REMS (Risk Evaluation and Mitigation Strategy) program enrollment due to hepatotoxicity risk; causes GI side effects (diarrhea, nausea) and fat-soluble vitamin (A, D, E, K) deficiencies requiring supplementation; must be taken with a low-fat diet (<20% fat) to minimize GI symptoms.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy