Juxtapid (lomitapide) / Novelion, Chiesi - LARVOL DELTA

Familial Hypercholesterolemia. (PubMed, Methodist Debakey Cardiovasc J) - "Prompt recognition and treatment with statins, often combined with ezetimibe, modifies the natural course of the disease...In statin-intolerant patients, bempedoic acid may provide additional therapeutic options. The most severe form, homozygous FH (HoFH), has an estimated prevalence of ~1 in 367,000 individuals and is associated with ASCVD in youth, calcific aortic stenosis, and a markedly reduced life expectancy. Patients with HoFH require specialized care and may need LDL apheresis and specific orphan drugs such as lomitapide or evinacumab."

Biomarker • Journal • Review • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Heterozygous Familial Hypercholesterolemia • Homozygous Familial Hypercholesterolemia • Metabolic Disorders • APOB

Durable lipid-lowering beyond 10 years with lomitapide in homozygous familial hypercholesterolemia: A real-world experience in Taiwan. (PubMed, J Clin Lipidol) - "Lomitapide provides durable LDL-C reduction beyond a decade with a manageable safety profile, supporting its long-term therapeutic role in East Asian patients with HoFH."

Journal • Real-world evidence • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders

Therapeutic challenges using lomitapide in patients with homozygous familial hypercholesterolemia in middle-income countries: analysis of three real-world cases. (ESC 2026) - No abstract available

Clinical • Real-world • Real-world evidence • Cardiovascular • Dyslipidemia

CDC20 exacerbates myocardial injury after myocardial infarction by regulating PPARα-mediated cardiomyocytes fatty acid metabolism (ESC 2026) - "CDC20 is a key regulator of metabolic dysfunction in cardiomyocytes after AMI. Lomitapide, which targets CDC20, may become a new and effective drug for treating AMI."

Cardiovascular • Congestive Heart Failure • Heart Failure • Myocardial Infarction • CDC20 • PPARA

Lipoprotein(a) carries triglyceride species associated with incident cardiovascular disease. (PubMed, Atherosclerosis) - "Native plasma Lp(a) carries a defined TG signature that is relatively stable postprandially and linked to incident cardiovascular disease."

Journal • Cardiovascular • Dyslipidemia • APOB • APOE

Long-term treatment of homozygous familial hypercholesterolemia with lomitapide: 11 years of safety and effectiveness findings from the Lomitapide Observational Worldwide Evaluation Registry. (PubMed, J Clin Lipidol) - P | "LOWER supports lomitapide's long-term effectiveness and safety for up to 11 years, enabling substantial LDL-C reductions and treatment goal achievement in HoFH with a manageable safety profile. Tentative evidence of reduced MACE during lomitapide treatment warrants further investigation."

Journal • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders

Therapeutic targeting of ROCK reverses EMT in lung cancer cells by impeding TAZ in Hippo signalling pathway. (PubMed, Mol Cell Biochem) - "At a lower IC50 than the controlled drug Fasudil, Lomitapide also induced increase in cellular ROS, mitochondrial membrane depolarization, G1/S phase cell cycle arrest with increase in apoptotic cell population. Reduction in migration, invasion, colony and sphere forming abilities also supported the annihilation of cancer progression. Overall, the therapeutic potency of Lomitapide was established to target Hippo pathway through disrupting ROCK activity in EMT dynamics."

Journal • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • CCND1 • MYC • TAFAZZIN

Virtual screening of Alpha-Aminoadipate Aminotransferase inhibitors from FDA approved drugs for Glutaric Acidemia Type I (SSIEM 2026) - "Conclusion Among the evaluated compounds, lomitapide and pimozide emerged as the most promising candidates, exhibiting both sustained binding stability during molecular dynamics simulations and the most favorable binding free energies under the MM/GBSA model. Further experimental studies are required to evaluate the inhibitory activity and therapeutic potential of these compounds in biochemical and cellular models."

CNS Disorders • Metabolic Disorders

Lomitapide as a Novel Therapeutic Agent in Breast Cancer: Mechanism and Potential Implications. (PubMed, Recent Pat Anticancer Drug Discov) - "Lomitapide is a novel repurposed agent for breast cancer treatment. ER-α and PPARγ are potential therapeutic targets."

Journal • Breast Cancer • Oncology • Solid Tumor • PPARG

Therapeutic targeting of ROCK reactivates Hippo Signalling and reverses EMT through impeding nuclear translocation of TAZ in lung cancer cells (EACR 2026) - "Nonetheless, at a lower IC50 than the controlled drug fasudil, lomitapide induced apoptosis along with a significant increase in cellular ROS, mitochondrial membrane depolarisation and G1/S phase cell cycle arrest. The therapeutic potency of lomitapide was established to target Hippo pathway through disrupting ROCK activity in EMT dynamics. The therapeutic potency of lomitapide was established to target Hippo pathway through disrupting ROCK activity in EMT dynamics. In LUAD cells, lomitapide effectively depleted TAZ, its oncogenic target, and reversed EMT. The treatment also triggered ROS-driven intrinsic apoptosis and cell cycle arrest."

Lung Cancer • Oncology • Solid Tumor • CCND1 • MYC • TAFAZZIN

Drugs for hypercholesterolemia. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal • Dyslipidemia • Metabolic Disorders

Computational Drug Repurposing Predicts FDA-Approved Drugs as Potential Inhibitors of Chikungunya Virus nsP2 Protease. (PubMed, J Phys Chem B) - "Subsequent molecular dynamics (MD) simulations revealed that, except for lomitapide, which dissociated from the pocket, the remaining leads formed dynamically stable complexes and modulated the catalytic environment in a chemotype-dependent manner. Among the small-molecule candidates, venetoclax and lapatinib emerge as computationally prioritized candidates for future biochemical and antiviral evaluation. Overall, this study provides mechanistic insight into noncovalent recognition of CHIKV nsP2pro and a framework for future experimental validation."

FDA event • Journal • Chikungunya • Infectious Disease

Drug repurposing for pancreatic cancer: Lomitapide Mesylate as a potent HK2 inhibitor discovered via virtual screening. (PubMed, Cell Signal) - "Pancreatic cancer has an early diagnosis rate below 5% and is largely resistant to conventional chemotherapy. Mechanistically, this synergy was linked to LM-mediated reversal of HK2-driven EMT. Thus, LM inhibits HK2 to block EMT and synergize with gemcitabine, offering a novel therapeutic strategy."

Journal • Oncology • Pancreatic Cancer • Solid Tumor • HK2

Homozygous familial hypercholesterolemia (HoFH) in Canada. (PubMed, Atherosclerosis) - "This analysis provides valuable insights into the evolving clinical profile of patients with HoFH across Canada. Despite advances in treatment, a significant proportion of patients continue to experience major cardiovascular events, underscoring the need for early diagnosis, aggressive lipid-lowering management, and equitable access to evidence-based therapies to optimize long-term outcomes and improve survival in this population."

Journal • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders • Rare Diseases

Chiesi Global Rare Diseases Announces European Commission Approval of LOJUXTA (lomitapide) Capsules for Paediatric Use in Homozygous Familial Hypercholesterolaemia (HoFH) (The Manila Times) - "The EC approval is based on evidence from a Phase 3, open-label, single-arm, multicentre study evaluating lomitapide in 43 pediatric participants aged 5 to 17 years with HoFH. The APH-19 study achieved its primary endpoint, demonstrating a mean 53.5% reduction in LDL‑C from baseline at week 24"

EMA approval • Homozygous Familial Hypercholesterolemia

New and Emerging Therapeutic Targets for ApoB-Containing Particles Lowering. (PubMed, Circ Res) - "Most established therapies that reduce major cardiovascular events, including statins, ezetimibe, PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors, and bempedoic acid, act primarily by enhancing LDL receptor-mediated clearance of apoB-containing particles...Lomitapide, an inhibitor of MTP (microsomal triglyceride transfer protein), and evinacumab, a monoclonal antibody targeting ANGPTL3 (angiopoietin-like protein 3), reduce LDL cholesterol in homozygous familial hypercholesterolemia by decreasing triglyceride-rich apoB-containing lipoproteins upstream of LDL particle formation...Gene-targeted approaches, including gene editing, epigenome editing, small interfering RNA, and antisense oligonucleotides, as well as novel oral or injectable agents and combination therapies, further broaden opportunities for durable apoB modulation. Transitioning from an LDL cholesterol-centric to an apoB-centric framework may represent a biologically integrated strategy to..."

Clinical • Journal • Review • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Hypertriglyceridemia • Metabolic Disorders • Pancreatitis • Severe Hypertriglyceridemia • ANGPTL3 • APOB

Loss of KLF15 expression characterizes proximal tubule injury in cisplatin-induced acute kidney injury: A multi-omics study. (PubMed, Curr Res Toxicol) - "Nephrotoxicity is a common side effect of cisplatin (CSP), a widely-used anti-tumor chemotherapy drug. Furthermore, virtual screening identified 6 drugs, including Simeprevir, Lomitapide, and Avodart, as potential high-affinity compounds targeting human KLF15. In conclusion, our study indicates that the downregulation of KLF15 is a prominent molecular feature in CSP-induced AKI, associated with metabolic failure and injury in proximal tubules."

Journal • Acute Kidney Injury • Nephrology • Oncology • Renal Disease • KLF5

Current and Future Perspectives of LDL-C Lowering Therapies 2026. (PubMed, J Atheroscler Thromb) - "Non-statin therapies, including ezetimibe, bile acid sequestrants, PCSK9 inhibitors, inclisiran, and bempedoic acid, are reviewed with an emphasis on their mechanisms, efficacy, and clinical positioning. Advanced therapies for severe dyslipidemia, such as lipoprotein apheresis, lomitapide, and evinacumab, are also discussed in this review...Collectively, these developments offer new opportunities to address unmet clinical needs, particularly in patients with FH, statin intolerance, and residual cardiovascular risk. A comprehensive understanding of these therapies is essential for further reducing the burden of ASCVD in the coming decades."

Journal • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Metabolic Disorders

LOWER: Lomitapide Observational Worldwide Evaluation Registry (clinicaltrials.gov) - P=N/A | N=260 | Completed | Sponsor: Amryt Pharma | Recruiting ➔ Completed | Trial completion date: Sep 2028 ➔ Apr 2026 | Trial primary completion date: Mar 2028 ➔ Apr 2026

Trial completion • Trial completion date • Trial primary completion date • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders

The evaluation of plozasiran for the treatment of familial chylomicronemia syndrome. (PubMed, Expert Rev Endocrinol Metab) - "We searched PubMed for all English language literature focusing on the search terms 'chylomicronemia,' 'hypertriglyceridemia,' 'APOC3 inhibition,' 'plozasiran,' 'olezarsen,' and 'volanesorsen.' We outline traditional management strategies and their limited role in FCS and explore non-traditional therapies including orlistat, lomitapide, inhibitors of angiopoietin like protein 3 (ANGPTL3), and analogues of fibroblast growth factor 21 (FGF21). In a phase 3 trial of plozasiran, at 10 months, median placebo-adjusted reductions in apo C-III were approximately -90%, while TG levels were reduced up to -59%. Thus, plozasiran and alternative RNA-based therapeutics directed against APOC3 represent transformational therapies for patients with FCS and related phenotypes characterized by severe recalcitrant hypertriglyceridemia."

Journal • Review • Dyslipidemia • Familial Chylomicronemia Syndrome • Hypertriglyceridemia • Pancreatitis • Severe Hypertriglyceridemia • ANGPTL3 • FGF21

Modulating IL-1β-induced pro-atherogenic endothelial responses through drug repurposing. (PubMed, Inflammopharmacology) - "Our findings highlight radotinib and lomitapide as promising repurposed small-molecule inhibitors of IL-1β signaling. By preserving endothelial integrity and dampening inflammatory responses, these compounds may serve as cost-effective and orally available alternatives to current biologic therapies. Further in vivo and mechanistic studies are needed to advance their potential clinical application."

Journal • Atherosclerosis • Cardiovascular • Inflammation • CDH5 • IL1B • IL1R1

MicroRNA-30c analog C2 decreases plasma cholesterol and atherosclerosis without causing liver injury in preclinical studies. (PubMed, Nat Commun) - "In contrast, MTP inhibitor lomitapide significantly reduced plasma lipids and caused hepatic steatosis. Mechanistic studies revealed that C2 reduced hepatic microsomal triglyceride transfer protein expression, secretion of apolipoprotein B-containing lipoproteins and FA synthesis and increased hepatic FA oxidation, plasma bile acids and fecal cholesterol excretion. C2 is a first-in-class microRNA therapeutic that decreases plasma cholesterol and atherosclerosis, without causing hepatic injury and inflammatory response."

Journal • Preclinical • Atherosclerosis • Cardiovascular • Diabetes • Dyslipidemia • Hepatology • Inflammation • Liver Failure • Metabolic Disorders • APOB • IL6 • MicroRNA 30c • TNFA

Inhibition of the SerpinB3/protease-activated receptor 2 axis reduces liver cancer development and affects lipid metabolism. (PubMed, Br J Cancer) - "1-PPA administration prevents lipid accumulation and HCC development in MASH-related liver carcinogenesis."

Journal • Fibrosis • Hepatocellular Cancer • Hepatology • Immunology • Liver Cancer • Metabolic Disorders • Metabolic Dysfunction-Associated Steatohepatitis • Metabolic Dysfunction-Associated Steatotic Liver Disease • Oncology • Solid Tumor • SERPINB3

Lomitapide mesylate and lomitapide target ALDOA to inhibit growth and enhance gemcitabine efficacy in PDAC. (PubMed, iScience) - "The observed synergistic or additive effects with gemcitabine depend on both specific PDAC cell line and chemical form of lomitapide, underscoring complexity of personalized combination therapies and need to consider drug properties and tumor biology. These findings support therapeutic repositioning of lomitapide mesylate and lomitapide for PDAC."

Journal • Oncology • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma • ALDOA

Lojuxta - opinion on variation to marketing authorisation (European Medicines Agency) - "On 26 March 2026, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a change to the terms of the marketing authorisation for the medicinal product Lojuxta...The CHMP adopted a change to the existing indication as follows: Lojuxta is indicated as an adjunct to a low‑fat diet and other lipid‑lowering medicinal products with or without low density lipoprotein (LDL) apheresis for the treatment of adult and paediatric patients aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH)....Genetic confirmation of HoFH should be obtained whenever possible. Other forms of primary hyperlipoproteinemia and secondary causes of hypercholesterolaemia (e.g. nephrotic syndrome, hypothyroidism) must be excluded."

CHMP • Homozygous Familial Hypercholesterolemia