Evkeeza (evinacumab-dgnb) / Regeneron, Ultragenyx - LARVOL DELTA

Evinacumab with or without lipoprotein apheresis in homozygous familial hypercholesterolaemia. (PubMed, Eur J Prev Cardiol) - "This study demonstrated considerable benefit of evinacumab for individuals with HoFH, with or without concurrent LA. Evinacumab appeared to lessen LA burden for some individuals. This analysis suggests that LA and evinacumab can be combined without compromising efficacy or safety."

Journal • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders • ANGPTL3

UTILIZATION, EXPENDITURE, AND PRICE TRENDS FOR EZETIMIBE AND NOVEL LIPID-LOWERING MEDICATIONS IN U.S. MEDICAID DRUG UTILIZATION DATA, 2002-2024 (ISPOR 2026) - "The analysis included ezetimibe (Zetia® and generic), and novel lipid-lowering medications: evolocumab (Repatha®), alirocumab (Praluent®), inclisiran (Leqvio®), bempedoic acid (Nexletol®), and evinacumab (Evkeeza®). Despite continued dominance of ezetimibe in prescription volume, Medicaid spending has shifted substantially toward high-cost NLLMs. These findings highlight a growing divergence between utilization and expenditure in Medicaid drug spending and underscore the importance of value-based pricing, outcomes-linked contracting, and formulary strategies as adoption of novel lipid-lowering agents expands."

Medicaid • Reimbursement • US reimbursement • ANGPTL3

STEPWISE COMBINATION THERAPY ACHIEVING SIGNIFICANT LDL-CHOLESTEROL REDUCTION IN SEVERE HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (ACC 2026) - "Decision-Making: The patient was started on rosuvastatin, ezetimibe, and evolocumab at his first visit in January 2024 while continuing biweekly apheresis (Fig. Stepwise combination therapy with statin, ezetimibe, PCSK9 inhibitor, evinacumab, and lomitapide achieved profound LDL-C reduction in a patient with severe HoFH due to a biallelic LDLR null variant, lowering pre-apheresis LDL-C levels by 95% with no safety or tolerability concerns."

Combination therapy • Coronary Artery Disease • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders • ANGPTL3

DOSE-RESPONSE RELATIONSHIP AND EFFICACY OF EVINACUMAB FOR REFRACTORY AND GENETIC DYSLIPIDEMIAS A SYSTEMATIC REVIEW AND META-ANALYSIS OF RANDOMIZED CONTROLLED TRIALS (ACC 2026) - "The 15mg/kg IV dose formulation of Evinacumab had an optimal dual lipid-lowering effect in the aforementioned dyslipidemia types where conventional therapies failed."

Retrospective data • Review • Dyslipidemia • Homozygous Familial Hypercholesterolemia • Hypertriglyceridemia • Metabolic Disorders • Mixed Hyperlipidemia

Update on familial hypercholesterolemia: An expert clinical consensus from the National Lipid Association. (PubMed, J Clin Lipidol) - "A stepwise approach to optimal therapy is outlined, beginning with lifestyle interventions and pharmacotherapy with maximally tolerated statins and ezetimibe. This update incorporates newer agents, including proprotein convertase subtilisin/kexin type 9 inhibitors and bempedoic acid. Additional therapies, such as lomitapide and evinacumab for homozygous FH and lipoprotein apheresis for heterozygous and homozygous FH, are discussed. Further topics include cardiovascular imaging for risk stratification, management in specific populations and circumstances, such as planning for and during pregnancy and in pediatrics, and recognition of health disparities. This guidance equips clinicians with evidence-based strategies to improve the identification and care of patients with FH, ultimately reducing the high morbidity and mortality associated with this condition."

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

Post-heparin plasma lipase activities in patients with severe hypertriglyceridemia treated with evinacumab. (PubMed, J Lipid Res) - "Post-heparin LPL triglyceride lipase activity was lower in patients with sHTG with bi-allelic LPL pathway mutations and increased in that group with evinacumab. The high variability in lipase activities observed via differing methods supports the need for more robust assays."

Journal • Dyslipidemia • Familial Chylomicronemia Syndrome • Hypertriglyceridemia • Severe Hypertriglyceridemia • ANGPTL3 • LPL

EVOLVE-HoFH: Assessing the Impact of Intensification of Lipid Lowering Therapy With Guidelines-based Evinacumab Administration on Coronary Plaque Volumes Measured by Coronary Computed Tomography Angiography (CCTA) in Patients With Homozygous Familial Hypercholesterolemia (HoFH) (clinicaltrials.gov) - P=N/A | N=52 | Not yet recruiting | Sponsor: Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)

New trial • Real-world evidence • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Metabolic Disorders

LDL-C target achievement after adding evinacumab in 2 patients with autosomal recessive hypercholesterolemia. (PubMed, J Clin Lipidol) - "These cases demonstrate a marked and clinically meaningful LDL-C-lowering effect of evinacumab in ARH, supporting its use as an effective LDL-R-independent therapeutic option."

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

Baseline characteristics and response to evinacumab in females and males with homozygous familial hypercholesterolemia in the ELIPSE OLE study. (PubMed, Am J Prev Cardiol) - "There was a trend, although not significant, toward higher relative precent decrease of LDL-C among females. In a study where half of the participants were females, evinacumab led to substantial LDL-C reduction in HoFH patients of both sexes, regardless of genotype or background LLT."

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

The 2025 ESC/EAS Dyslipidaemia Focused Update: Clinical Interpretation and Practical Implications. (PubMed, Cardiovasc Drugs Ther) - "New evidence supporting bempedoic acid, inclisiran and evinacumab expands the therapeutic armamentarium, particularly for patients at high, very high and extreme cardiovascular risk or those with statin intolerance. In the acute coronary syndrome (ACS) setting, the update reinforces a more proactive, early-intensification approach. Overall, the Focused Update refines the operational framework of LDL-C management, promoting earlier, more personalized and more sustained lipid lowering to reduce cumulative atherosclerotic exposure."

Journal • Review • Acute Coronary Syndrome • Atherosclerosis • Cardiovascular • Dyslipidemia

From physiopathology to treatment of familial hypercholesterolemia: Existing and emerging pharmacotherapies. (PubMed, Pharmacol Rev) - "This includes established drugs such as proprotein convertase subtilisin/kexin type 9 inhibitors, inclisiran, lomitapide, and bempedoic acid. Emerging therapies include evinacumab, lerodalcibep, antisense oligonucleotide-based drugs, certain cholesteryl ester transfer protein inhibitors like obicetrapib, AZD8233, gemcabene, diacylglycerol O-acyltransferase-2 inhibitors, acyl-CoA:cholesterol acyltransferase-2 inhibitors, vupanorsen, volanesorsen, olezarsen, pelacarsen (TQJ230), olpasiran (AMG890), zerlasiran (SLN360), lepodisiran (LY3819469), and muvalaplin...Recent pharmacological advancements provide significant opportunities for successful low-density lipoprotein cholesterol management and control of FH. Although some of these agents are already used, several highly effective compounds are in development, heralding a promising future for FH treatment."

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

Advanced therapy in familial hypercholesterolemia. (PubMed, Can Fam Physician) - "FH is a complex genetic disorder causing highly elevated levels of LDL-C and risk of cardiovascular disease. Any child with homozygous FH should be referred to a specialist for consultation. Monoclonal antibodies are recommended by guidelines as adjunct treatments for FH when statins are insufficient. They have been studied as treatment for adults with FH, but direct evidence for pediatric patients is limited. Proprotein convertase subtilisin-kexin type 9 (PCSK9) inhibitors and angiopoietin-like protein 3 (ANGPTL3) inhibitors can provide meaningful reductions in LDL-C levels when conventional therapies alone, including statins and ezetimibe, are inadequate. Evolocumab, a PCSK9 inhibitor, has demonstrated safety and efficacy in adult patients with heterozygous FH and a moderate effect in children with homozygous FH. Evinacumab, an ANGPTL3 inhibitor, has shown greater efficacy than evolocumab for treating homozygous FH in limited pediatric studies. Longer-term and..."

Journal • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Heterozygous Familial Hypercholesterolemia • Homozygous Familial Hypercholesterolemia • Metabolic Disorders • Pediatrics • ANGPTL3

Efficacy of evinacumab by genotype and low-density lipoprotein receptor function in patients with homozygous familial hypercholesterolaemia: A subanalysis from the ELIPSE open-label extension study. (PubMed, Atherosclerosis) - "Evinacumab treatment resulted in sustained LDL-C reduction in patients with HoFH irrespective of genotype or LDLR function."

Journal • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • ANGPTL3 • LDLR

ANCHOR-POC: Study to Assess the Effects of Angiopoietin-like Protein 3 (ANGPTL3) Inhibition in Adult Participants With Diabetic Kidney Disease (clinicaltrials.gov) - P2 | N=270 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Not yet recruiting ➔ Recruiting | Trial completion date: Dec 2027 ➔ Apr 2028 | Trial primary completion date: Jun 2027 ➔ Sep 2027

Enrollment open • Trial completion date • Trial primary completion date • Diabetic Nephropathy • Nephrology • Renal Disease • APOB

Seventeen years to change practice: will the 2025 ESC/EAS dyslipidaemia guidelines finally break the Sisyphean cycle? (PubMed, Atherosclerosis) - "The extension of the pharmacological arsenal of lipid-lowering therapies with bempedoic acid, evinacumab, inclisiran, icosapent ethyl (and not mixtures with of eicosapentaenoic acid and docosahexaenoic acid) have enriched the arsenal of LDL-cholesterol and triglyceride-lowering drugs...Therefore, the guidelines recommend immediate initiation of high-intensity statin with ezetimibe for most patients with acute coronary syndrome or the immediate intensification of the therapy in those who are already on lipid-lowering drugs at the acute event...This update also has weaknesses, including missed opportunities to promote a more courageous approach for combination therapies, limitations in the diabetes risk classification, and discrepancies across ESC guideline papers which hopefully will be overcome in the next full update. Since real-world clinical adoption is far slower than evidence creation, implementation lags remain a Sisyphean obstacle, highlighting the ongoing need..."

Journal • Review • Acute Coronary Syndrome • Cardiovascular • Diabetes • Dyslipidemia • Metabolic Disorders

Angiopoietin-like Protein 3 (ANGPTL3) Targeting in the Management of Dyslipidemias. (PubMed, Int J Mol Sci) - "We also discuss Evinacumab, a monoclonal antibody, its structure, mechanism of action, safety, tolerability, pharmacokinetics, and pharmacodynamics, as well as its clinical trial-derived results. The antisense oligonucleotides modify ANGPTL3 mRNA to inhibit protein production, and small interfering RNAs induce mRNA degradation; results from clinical trials were reviewed in detail. Finally, we discuss promising gene editing approaches including clustered regularly interspaced short palindromic repeats (CRISPR)/Cas systems."

Journal • Review • Cardiovascular • Dyslipidemia • Metabolic Disorders • ANGPTL3

The ANGPTL3-integrin α5 axis drives retinal vascular leakage in diabetic retinopathy. (PubMed, J Transl Med) - No abstract available

Journal • Diabetic Retinopathy • Retinal Disorders • ANGPTL3

Evinacumab in patients aged 5-17 years with homozygous familial hypercholesterolemia. (PubMed, Atherosclerosis) - P3 | "Evinacumab markedly reduced LDL-C in children and adolescents with HoFH, beyond optimized standard LLT and lipoprotein apheresis. LDL-C remains above goal in most pediatric patients with HoFH, and evinacumab should be routinely considered whenever further LDL-C lowering is needed."

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

Authors' Reply to Khan et al. "Evaluating the Effectiveness and Safety of Evinacumab in Treating Hypercholesterolemia and Hypertriglyceridemia: A Systematic Review and Meta-analysis of Randomized Controlled Trials". (PubMed, Am J Cardiovasc Drugs) - No abstract available

Journal • Retrospective data • Dyslipidemia • Hypertriglyceridemia • Metabolic Disorders

Comment on "Evaluating the Effectiveness and Safety of Evinacumab in Treating Hypercholesterolemia and Hypertriglyceridemia: A Systematic Review and Meta-analysis of Randomized Controlled Trials". (PubMed, Am J Cardiovasc Drugs) - No abstract available

Journal • Retrospective data • Dyslipidemia • Hypertriglyceridemia • Metabolic Disorders

Pharmacists role in attaining evinacumab for use in patients with persistent hypercholesterolemia (ASHP 2025) - No abstract available

Clinical • Dyslipidemia • Metabolic Disorders

Achieving the impossible: effective reduction of low-density lipoprotein cholesterol (LDL-C) in a patient with homozygous familial hypercholesterolemia. (PubMed, Endokrynol Pol) - "Not required for Clinical Vignette."

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

ANCHOR-POC: Study to Assess the Effects of Angiopoietin-like Protein 3 (ANGPTL3) Inhibition in Adult Participants With Diabetic Kidney Disease (clinicaltrials.gov) - P2 | N=270 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals

New P2 trial • Diabetic Nephropathy • Nephrology • Renal Disease • APOB

Therapeutic Plasma Exchange and Evinacumab for Homozygous Familial Hypercholesterolemia. (PubMed, JACC Case Rep) - "Persistence and use of combination therapies are required in treating HoFH, especially when patients experience intolerance to commonly used treatments. Premedications can prevent allergic and allergy-like reactions to therapies in HoFH."

Journal • Allergy • Atherosclerosis • Cardiovascular • Dyslipidemia • Familial Hypercholesterolemia • Genetic Disorders • Homozygous Familial Hypercholesterolemia • Immunology • Metabolic Disorders • ANGPTL3

Effects of evinacumab on high-density lipoprotein function in patients with homozygous familial hypercholesterolemia. (PubMed, J Clin Lipidol) - "HDL function assessed by serum CUC level increased by evinacumab in patients with genetically diagnosed HoFH, although the change of CUC was correlated with that of HDL cholesterol level."

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