Lomitapide (Juxtapid): how it works, status and trials
Approved 2012 (HoFH only)PillLowering LDL cholesterol
Lomitapide (Juxtapid) is taken by mouth daily. It keeps the liver and gut from loading fat onto apoB, so they release fewer fat-carrying particles, and it works without LDL receptors. It is approved only for homozygous familial hypercholesterolemia (HoFH), through a restricted program, and can cause liver fat and stomach side effects.
In technical terms: Blocks loading of lipid onto apoB, so less VLDL and chylomicron is secreted. LDL-receptor independent; liver fat and GI effects.
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At a glance
- Acts on
- MTP · liver and intestine
- How it is given
- Oral · daily (restricted programme)
- What the main trial found
- LDL-C ≈ −50% in HoFH (−53.5% in children)
- Status (curated October 25, 2026)
- Approved 2012 (HoFH only)
Where it fits in the body
Triglyceride-rich lipoproteins
VLDL from the liver and chylomicrons from the gut carry triglyceride. Lipoprotein lipase (LPL) on capillary walls breaks it down. ANGPTL3 and apoC-III act as brakes on this process. Removing either brake (11–15) lowers triglycerides, and ANGPTL3 blockade also lowers LDL-C without needing LDL receptors, which matters in homozygous FH.
Evidence
- Paediatric HoFH (PubMed 39426393)
Common questions
How does Lomitapide (Juxtapid) work?
Lomitapide (Juxtapid) is taken by mouth daily. It keeps the liver and gut from loading fat onto apoB, so they release fewer fat-carrying particles, and it works without LDL receptors. It is approved only for homozygous familial hypercholesterolemia (HoFH), through a restricted program, and can cause liver fat and stomach side effects. In technical terms: Blocks loading of lipid onto apoB, so less VLDL and chylomicron is secreted. LDL-receptor independent; liver fat and GI effects. It acts on MTP · liver and intestine.
How much does Lomitapide (Juxtapid) lower cholesterol or other lipids?
LDL-C ≈ −50% in HoFH (−53.5% in children). Individual results differ, and a clinician can say what to expect for one person.
Is Lomitapide (Juxtapid) approved, and what is the status?
Approved 2012 (HoFH only) (curated October 25, 2026).
How is Lomitapide (Juxtapid) taken?
Oral · daily (restricted programme).
Read more
Related therapies
- Statins: Approved (generic)
- Bempedoic acid (Nexletol, Nexlizet): Approved 2020
- Ezetimibe (Zetia): Approved (generic)
- Bile acid sequestrants (colesevelam): Approved (generic)
Educational, not medical advice. Mechanism and status text curated October 25, 2026; registry data refreshed October 27, 2026. Registries can lag company announcements. Effects are placebo-adjusted results from the main published trial, not head-to-head comparisons. Investigational agents are available only in clinical trials. Preval Health does not practice medicine; licensed clinicians in a partner network provide care. If you have chest pain or signs of a stroke, call 911.