ArticleCore evidence2019
Lomitapide: a review of its clinical use, efficacy, and tolerability.
Article in Core evidence, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 35 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
35 citing papers in PubMed, 1 synthesis or guideline pooled it, 73 citations in OpenAlex.
- Familial hypercholesterolemia - treatment update in children, systematic review.Pediatric endocrinology, diabetes, and metabolism · 2022Pooled it
- Evinacumab in homozygous familial hypercholesterolaemia: long-term safety and efficacy.European heart journal · 2024Trial
- A whole-animal phenotypic drug screen identifies suppressors of atherogenic lipoproteins.eLife · 2026Article
- Familial Hypercholesterolemia in Women: Diagnosis, Treatment, and Cardiovascular Outcomes Across the Lifespan.Current atherosclerosis reports · 2026Review
- Lomitapide mesylate and lomitapide target ALDOA to inhibit growth and enhance gemcitabine efficacy in PDAC.iScience · 2026Article
- Acyl-CoA Synthetase 5 Knockout and Inhibitors Protect Against Diet-Induced Obesity in Mice by Activating the Ileal Brake.Journal of the Endocrine Society · 2026Article
- Discovery of Lomitapide as a Novel Mast Cell Regulatory Agent Through Chemical Library Screening.European journal of immunology · 2026Article
- Adaptation to cystine limitation stress confers a targetable lipid metabolism vulnerability in pancreatic ductal adenocarcinoma.Nature communications · 2025Article
- Using chanarin-dorfman syndrome patient fibroblasts to explore disease mechanisms and new treatment avenues.Orphanet journal of rare diseases · 2025Article
- Unveiling the crossroads of STING signaling pathway and metabolic reprogramming: the multifaceted role of the STING in the TME and new prospects in cancer therapies.Cell communication and signaling : CCS · 2025Review
- Article
- Review
- Expression and prognostic significance of microsomal triglyceride transfer protein in brain tumors: a retrospective cohort study.Translational cancer research · 2024Article
- Lipoprotein a - Lp(a).Indian heart journal · 2024Review
- Established and Emerging Lipid-Lowering Drugs for Primary and Secondary Cardiovascular Prevention.American journal of cardiovascular drugs : drugs, devices, and other interventions · 2023Review
- Intracellular Cholesterol Trafficking.Cold Spring Harbor perspectives in biology · 2023Review
- The Beneficial Effect of Lomitapide on the Cardiovascular System in LDLrAntioxidants (Basel, Switzerland) · 2023Article
- Gene transfer and genome editing for familial hypercholesterolemia.Frontiers in molecular medicine · 2023Review
- Current Understanding on the Role of Lipids in Macrophages and Associated Diseases.International journal of molecular sciences · 2022Review
- Familial Hypercholesterolemia and Its Current Diagnostics and Treatment Possibilities: A Literature Analysis.Medicina (Kaunas, Lithuania) · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Lomitapide is an inhibitor of MTP, an enzyme located in the endoplasmic reticulum of hepatocytes and enterocytes. This enzyme is responsible for the synthesis of very low-density lipoproteins in the liver and chylomicrons in the intestine. Lomitapide has been approved by the US Food and Drug Administration, European Medicines Agency, and other regulatory agencies for the treatment of hypercholesterolemia in adult patients with homozygous familial hypercholesterolemia. Clinical trials have shown that lomitapide reduces low-density-lipoprotein cholesterol levels by around 40% in homozygous familial hypercholesterolemia patients on treatment with statins with or without low-density-lipoprotein apheresis, with an acceptable safety and tolerance profile. The most common adverse events are gastrointestinal symptoms that decrease in frequency with long-term treatment, and the increase in liver fat remains stable. This review analyzes the clinical use, efficacy, and tolerability of lomitapide.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.