Drug · FAERS side-effect profile with label screen

Lomitapide side effects: what 3,759 FDA adverse event reports show

Lomitapide Mesylate · 85 reaction terms · 7,363 drug–reaction co-reports · data 2026-09-14

Drug class

Microsomal triglyceride transfer protein inhibitor

FDA label, Indications wording of the closest related product

Label matched

Lomitapide Mesylate (Juxtapid)

closest FDA label found for this product string; brand list suppressed because the label is a related product, not an exact match

Who reports

3,759 reports · 33% serious · 94 with death outcome · 65% female

openFDA FAERS counts, all years, deduplication not applied

Used for

JUXTAPID 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).

FDA label, Indications and Usage

What it is

JUXTAPID capsules contain lomitapide mesylate, a synthetic lipid-lowering agent for oral administration.

FDA label, Description section

Boxed 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).

FDA label

How to read this page. FAERS is a spontaneous-reporting system: counts are not incidence rates and a signal is not proof of causation. Each reaction below is run through an automated screen — is the term in the FDA label (known), in the indications (the disease being treated), an administrative/coding term, or none of these (unlabeled)? That screen is mechanical text matching, not a causality assessment. The 30 hand-written Bradford Hill assessments are on the causality assessment page. Not medical advice.

Causality screen of the 40 strongest signals

Labeled — boxed warning 6Labeled adverse reaction 11Labeled (related wording) 5Reporting / coding artifact 6Lab value / disease marker 1Not in label — unassessed 11

What the badges mean

Labeled — boxed warning The term (or its clinical synonym) appears in the FDA boxed warning: causality is regulator-recognised and considered serious.
Labeled adverse reaction The exact term appears in the label's warnings or adverse-reactions sections: a known, expected effect. The FAERS signal confirms the label rather than discovering anything.
Labeled (related wording) The label describes this effect under different wording (e.g. 'neutropenia' for 'neutrophil count decreased'). Treated as known.
Reporting / coding artifact An administrative, monitoring or exposure term (product issue, off-label use, 'test normal', overdose...). Not a biological reaction; a high PRR here reflects how reports are coded.
Lab value / disease marker A laboratory result, investigation or disease-activity marker (antibody positive, CRP abnormal, swollen joint count) not described in the label. These usually mirror the monitored disease or testing protocol rather than a drug effect; not screened further.
Not in label — unassessed Not found in the label text, not an artifact pattern. This is a genuine candidate for a formal causality assessment (Bradford Hill / WHO-UMC); it has NOT had one here.

Strongest disproportionality signals

Flagged terms (PRR ≥ 2, χ² ≥ 4, n ≥ 10), ranked by PRR; infinite PRRs (no comparator reports) excluded. The EBGM column is a second, shrinkage-based estimate (MGPS) with its 5th percentile EB05 and the BCPNN IC025; a pair flagged by PRR alone is marked PRR-only and should be read as fragile. The strata column recomputes the PRR on serious reports only and flags pairs whose reports are unusually skewed by sex or come mostly from consumers rather than clinicians. 45 further terms are in the interactive explorer. Reaction links open the term's cross-drug page.

Show: hide coding/lab/outcome terms hide indication terms unlabeled only hide PRR-only

Reaction	Reports	PRR 95% CI · ROR · χ²/p	EBGM EB05 · IC025	Label screen	Serious-only & strata	Trial cross-check
Therapy cessation	792	87.8 82.5–93.5ROR 111.0 · χ² 66,997 / p <1e-300	86.3 EB05 81.4 · IC025 +6.2 3/3 methods agree	Reporting / coding artifact	serious PRR 169.8	no trial data cached
Flatulence (gas)	241	23.9 21.1–27.0ROR 25.4 · χ² 5,245 / p <1e-300	23.7 EB05 21.3 · IC025 +4.3 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 27.7	no trial data cached
Liver function test increased	73	19.2 15.3–24.1ROR 19.5 · χ² 1,235 / p 8e-271	19.0 EB05 15.6 · IC025 +3.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 34.5	no trial data cached
Blood cholesterol increased	155	18.6 15.9–21.7ROR 19.4 · χ² 2,560 / p <1e-300	18.5 EB05 16.2 · IC025 +3.9 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 14.1	no trial data cached
Hepatic steatosis	61	18.3 14.3–23.5ROR 18.6 · χ² 981 / p 1e-215	18.2 EB05 14.6 · IC025 +3.6 3/3 methods agree	Labeled — boxed warning exact term	serious PRR 33.1	no trial data cached
Liver function test abnormal	94	18.0 14.7–21.9ROR 18.4 · χ² 1,487 / p <1e-300	17.9 EB05 15.0 · IC025 +3.7 3/3 methods agree	Reporting / coding artifact	serious PRR 26.4	no trial data cached
Weight decreased (weight loss)	842	16.9 15.9–17.9ROR 21.5 · χ² 12,695 / p <1e-300	16.8 EB05 15.9 · IC025 +3.9 3/3 methods agree	Labeled (related wording) related wording	serious PRR 11.0	no trial data cached
Weight fluctuation	29	15.6 10.8–22.4ROR 15.7 · χ² 380 / p 6e-85	15.2 EB05 10.9 · IC025 +3.0 3/3 methods agree	Not in label — unassessed	serious n=4	no trial data cached
Hepatic enzyme increased	188	15.5 13.5–17.8ROR 16.2 · χ² 2,532 / p <1e-300	15.4 EB05 13.6 · IC025 +3.7 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 26.0	no trial data cached
Gastroenteritis viral	47	14.5 10.9–19.3ROR 14.7 · χ² 577 / p 9e-128	14.3 EB05 11.1 · IC025 +3.2 3/3 methods agree	Not in label — unassessed	serious PRR 13.4	no trial data cached
Inappropriate schedule of drug administration	184	14.3 12.4–16.4ROR 14.9 · χ² 2,257 / p <1e-300	14.2 EB05 12.5 · IC025 +3.5 3/3 methods agree	Reporting / coding artifact	serious PRR 29.4	no trial data cached
Alanine aminotransferase increased	147	13.4 11.4–15.7ROR 13.9 · χ² 1,672 / p <1e-300	13.3 EB05 11.6 · IC025 +3.4 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 21.7	no trial data cached
Aspartate aminotransferase increased	114	12.0 10.0–14.4ROR 12.3 · χ² 1,139 / p 6e-250	11.9 EB05 10.1 · IC025 +3.2 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 19.9	no trial data cached
Blood triglycerides increased	37	11.7 8.5–16.1ROR 11.8 · χ² 350 / p 2e-78	11.2 EB05 8.3 · IC025 +2.8 3/3 methods agree	Lab value / disease marker	serious PRR 10.5	no trial data cached
Diarrhoea (diarrhea)	1,069	9.3 8.8–9.8ROR 12.6 · χ² 8,131 / p <1e-300	9.3 EB05 8.8 · IC025 +3.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 8.6	no trial data cached
Abdominal discomfort	239	8.0 7.1–9.0ROR 8.5 · χ² 1,463 / p <1e-300	7.9 EB05 7.1 · IC025 +2.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 8.5	no trial data cached
Gastrointestinal disorder	125	7.9 6.6–9.4ROR 8.1 · χ² 748 / p 5e-165	7.7 EB05 6.6 · IC025 +2.6 3/3 methods agree	Labeled (related wording) related wording	serious PRR 6.6	no trial data cached
Abdominal pain upper	284	7.7 6.9–8.6ROR 8.3 · χ² 1,669 / p <1e-300	7.6 EB05 6.9 · IC025 +2.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 9.5	no trial data cached
Angina pectoris	36	7.0 5.0–9.6ROR 7.0 · χ² 178 / p 6e-41	6.5 EB05 4.9 · IC025 +2.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 11.3	no trial data cached
Hepatomegaly	12	6.8 3.9–11.9ROR 6.8 · χ² 54 / p 1e-13	5.5 EB05 3.3 · IC025 +1.5 3/3 methods agree	Not in label — unassessed	serious PRR 10.8	no trial data cached
Eructation (belching)	23	6.8 4.5–10.2ROR 6.8 · χ² 107 / p 2e-25	6.0 EB05 4.2 · IC025 +1.9 3/3 methods agree	Not in label — unassessed	serious n=10	no trial data cached
Abdominal distension (bloating)	125	6.7 5.6–8.0ROR 6.9 · χ² 604 / p 9e-134	6.6 EB05 5.6 · IC025 +2.4 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 6.5	no trial data cached
Drug dose omission	184	6.1 5.3–7.1ROR 6.4 · χ² 790 / p 5e-174	6.0 EB05 5.3 · IC025 +2.4 3/3 methods agree	Reporting / coding artifact	serious PRR 11.8	no trial data cached
Gastrointestinal pain	12	6.1 3.5–10.8ROR 6.1 · χ² 46 / p 5e-12	5.1 EB05 3.1 · IC025 +1.4 3/3 methods agree	Labeled (related wording) related wording	serious PRR 8.2	no trial data cached
Coronary artery occlusion	15	6.1 3.7–10.1ROR 6.1 · χ² 59 / p 1e-14	5.2 EB05 3.3 · IC025 +1.5 3/3 methods agree	Not in label — unassessed	serious PRR 10.7	no trial data cached
International normalised ratio increased	28	5.4 3.8–7.9ROR 5.5 · χ² 97 / p 4e-23	5.0 EB05 3.6 · IC025 +1.7 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 7.6	no trial data cached
Dyspepsia (indigestion)	95	5.4 4.4–6.6ROR 5.5 · χ² 338 / p 9e-76	5.3 EB05 4.4 · IC025 +2.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 5.2	no trial data cached
Hunger	13	5.4 3.1–9.3ROR 5.4 · χ² 42 / p 4e-11	4.6 EB05 2.9 · IC025 +1.3 3/3 methods agree	Not in label — unassessed	serious n=2	no trial data cached
Renal pain	10	5.3 2.8–9.8ROR 5.3 · χ² 31 / p 2e-8	4.3 EB05 2.5 · IC025 +1.1 3/3 methods agree	Not in label — unassessed	serious PRR 4.1	no trial data cached
Treatment noncompliance	51	5.2 4.0–6.9ROR 5.3 · χ² 171 / p 2e-39	5.0 EB05 4.0 · IC025 +1.9 3/3 methods agree	Reporting / coding artifact	serious PRR 5.6	no trial data cached
Constipation	168	4.3 3.7–5.0ROR 4.5 · χ² 432 / p 4e-96	4.3 EB05 3.8 · IC025 +1.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 4.4	no trial data cached
Gastrooesophageal reflux disease (acid reflux)	60	4.2 3.2–5.4ROR 4.2 · χ² 142 / p 4e-33	4.1 EB05 3.3 · IC025 +1.6 3/3 methods agree	Not in label — unassessed	serious PRR 3.7	no trial data cached
Faeces discoloured	16	4.1 2.5–6.7ROR 4.1 · χ² 35 / p 2e-9	3.7 EB05 2.5 · IC025 +1.1 3/3 methods agree	Not in label — unassessed	serious PRR 5.5	no trial data cached
Influenza	80	4.0 3.2–5.0ROR 4.1 · χ² 177 / p 9e-41	3.9 EB05 3.2 · IC025 +1.6 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 3.4	no trial data cached
Laboratory test abnormal	21	4.0 2.6–6.1ROR 4.0 · χ² 44 / p 2e-11	3.7 EB05 2.6 · IC025 +1.2 3/3 methods agree	Reporting / coding artifact	serious PRR 2.4	no trial data cached
Abdominal pain	165	4.0 3.4–4.6ROR 4.1 · χ² 366 / p 7e-82	3.9 EB05 3.4 · IC025 +1.7 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 5.0	no trial data cached
Chromaturia	16	4.0 2.4–6.4ROR 4.0 · χ² 32 / p 6e-9	3.6 EB05 2.4 · IC025 +1.0 3/3 methods agree	Not in label — unassessed	serious PRR 4.1	no trial data cached
Nausea	556	3.9 3.6–4.2ROR 4.4 · χ² 1,260 / p 3e-276	3.9 EB05 3.7 · IC025 +1.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 5.1	no trial data cached
Frequent bowel movements	18	3.7 2.4–5.9ROR 3.7 · χ² 33 / p 4e-9	3.4 EB05 2.3 · IC025 +1.0 3/3 methods agree	Not in label — unassessed	serious PRR 3.4	no trial data cached
Liver injury	15	3.5 2.1–5.7ROR 3.5 · χ² 24 / p 5e-7	3.2 EB05 2.1 · IC025 +0.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 6.3	no trial data cached

Trial evidence (ClinicalTrials.gov)

Trial cross-check not yet run for this drug (ClinicalTrials.gov pull covers the 800 most-reported drugs first).

Most frequently reported reactions

By raw co-report count, regardless of disproportionality. Common terms here are often the illness itself or generic outcomes.

Reaction	Reports	PRR 95% CI · ROR · χ²/p	EBGM EB05 · IC025	Label screen	Serious-only & strata	Trial cross-check
Diarrhoea (diarrhea)	1,069	9.3 8.8–9.8ROR 12.6 · χ² 8,131 / p <1e-300	9.3 EB05 8.8 · IC025 +3.1 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 8.6	no trial data cached
Weight decreased (weight loss)	842	16.9 15.9–17.9ROR 21.5 · χ² 12,695 / p <1e-300	16.8 EB05 15.9 · IC025 +3.9 3/3 methods agree	Labeled (related wording) related wording	serious PRR 11.0	no trial data cached
Therapy cessation	792	87.8 82.5–93.5ROR 111.0 · χ² 66,997 / p <1e-300	86.3 EB05 81.4 · IC025 +6.2 3/3 methods agree	Reporting / coding artifact	serious PRR 169.8	no trial data cached
Nausea	556	3.9 3.6–4.2ROR 4.4 · χ² 1,260 / p 3e-276	3.9 EB05 3.7 · IC025 +1.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 5.1	no trial data cached
Abdominal pain upper	284	7.7 6.9–8.6ROR 8.3 · χ² 1,669 / p <1e-300	7.6 EB05 6.9 · IC025 +2.7 3/3 methods agree	Labeled (related wording) related wording	serious PRR 9.5	no trial data cached
Flatulence (gas)	241	23.9 21.1–27.0ROR 25.4 · χ² 5,245 / p <1e-300	23.7 EB05 21.3 · IC025 +4.3 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 27.7	no trial data cached
Abdominal discomfort	239	8.0 7.1–9.0ROR 8.5 · χ² 1,463 / p <1e-300	7.9 EB05 7.1 · IC025 +2.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 8.5	no trial data cached
Hepatic enzyme increased	188	15.5 13.5–17.8ROR 16.2 · χ² 2,532 / p <1e-300	15.4 EB05 13.6 · IC025 +3.7 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 26.0	no trial data cached
Drug dose omission	184	6.1 5.3–7.1ROR 6.4 · χ² 790 / p 5e-174	6.0 EB05 5.3 · IC025 +2.4 3/3 methods agree	Reporting / coding artifact	serious PRR 11.8	no trial data cached
Inappropriate schedule of drug administration	184	14.3 12.4–16.4ROR 14.9 · χ² 2,257 / p <1e-300	14.2 EB05 12.5 · IC025 +3.5 3/3 methods agree	Reporting / coding artifact	serious PRR 29.4	no trial data cached
Constipation	168	4.3 3.7–5.0ROR 4.5 · χ² 432 / p 4e-96	4.3 EB05 3.8 · IC025 +1.8 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 4.4	no trial data cached
Abdominal pain	165	4.0 3.4–4.6ROR 4.1 · χ² 366 / p 7e-82	3.9 EB05 3.4 · IC025 +1.7 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 5.0	no trial data cached
Blood cholesterol increased	155	18.6 15.9–21.7ROR 19.4 · χ² 2,560 / p <1e-300	18.5 EB05 16.2 · IC025 +3.9 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 14.1	no trial data cached
Alanine aminotransferase increased	147	13.4 11.4–15.7ROR 13.9 · χ² 1,672 / p <1e-300	13.3 EB05 11.6 · IC025 +3.4 3/3 methods agree	Labeled — boxed warning synonym match	serious PRR 21.7	no trial data cached
Abdominal distension (bloating)	125	6.7 5.6–8.0ROR 6.9 · χ² 604 / p 9e-134	6.6 EB05 5.6 · IC025 +2.4 3/3 methods agree	Labeled adverse reaction exact term	serious PRR 6.5	no trial data cached

Unlabeled candidate signals

These flagged terms were not found in the FDA label text and do not match an artifact pattern. They are the pairs that would merit a structured assessment (temporality, dose-response, biological plausibility, consistency across databases, trial or cohort evidence). The screen has not done that; string-matching misses label wording, and a strong PRR can still be stimulated reporting, co-medication, or the disease itself.

Weight fluctuation — n=29, PRR 15.6
Gastroenteritis viral — n=47, PRR 14.5
Eructation — n=23, PRR 6.8
Gastrooesophageal reflux disease — n=60, PRR 4.2

Want to assess one of these properly? The runbook describes the Bradford Hill / trial-evidence workflow used on the assessment page, and handoff_top1000_signals.csv lists the strongest unassessed pairs across all drugs.

Common questions

What are the most reported side effects of Lomitapide?

In FDA FAERS, the most frequently co-reported reaction terms for Lomitapide are diarrhoea, weight decreased, nausea, abdominal pain upper, flatulence, abdominal discomfort. These are report counts, not rates: FAERS has no denominator, reporting is voluntary and uneven, and a term can be reported because of the illness being treated rather than the drug.

Does Lomitapide have an FDA boxed warning?

Yes. The current FDA label carries a boxed 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).

Do FAERS signals prove that Lomitapide causes these side effects?

No. A disproportionality signal (PRR, ROR) means a reaction is reported more often with this drug than with others in the database. That can reflect a real effect, but also the disease being treated, stimulated reporting after news or litigation, co-medications, or how reports are coded. Causality needs temporality, dose-response, biological plausibility, consistency across data sources and, ideally, trial or cohort evidence. This page screens each term against the FDA label and ClinicalTrials.gov results; it does not establish causation.

Sources & methods

FDA label via openFDA (set ID 61a0be9d-ee58-43e0-b5cb-141011835081, effective 20260330; matched on openfda.generic_name.exact:"LOMITAPIDE MESYLATE")
Bayesian shrinkage measures computed offline over all 1,317,300 cached pairs: BCPNN information component (IC, with Norén closed-form IC025) and MGPS empirical-Bayes geometric mean (EBGM, EB05) with a two-component gamma prior fitted by maximum marginal likelihood (α₁=0.57, β₁=0.02, α₂=1.42, β₂=0.70, w=0.17); unstratified
FAERS pair statistics from the OpenPV build of 2026-09-14 (openFDA count= aggregation, field patient.drug.openfda.generic_name.exact, 85 reaction terms, min 3 co-reports); PRR/ROR with 95% CI, chi-square with Yates correction
Serious-only and stratified counts (serious:1, patient.patientsex, primarysource.qualification) from openFDA count queries; serious-only PRR uses the drug serious report total and the term global serious total (N_serious=11,882,979); strata shown only when n ≥ 20
Seriousness and sex counts from openFDA count=serious, patient.patientsex, seriousnessdeath

Page generated 2026-10-08. Label and PubChem text are retrieved automatically and may lag the current label; always check the current prescribing information. OpenPV is a beta research tool of the Open Source Medicine Foundation.