Evinacumab for treatment of familial hypercholesterolemia

References

• Gidding SS, Champagne MA, de Ferranti SD, et al. The agenda for familial hypercholesterolemia: a scientific statement from the American heart association. Circulation. 2015 Dec 1;132(22):2167–2192.
   PubMed Web of Science ®Google Scholar
• Beheshti SO, Madsen CM, Varbo A, et al. Worldwide prevalence of familial hypercholesterolemia: meta-analyses of 11 million subjects. J Am Coll Cardiol. 2020 May 26;75(20):2553–2566.
   PubMed Web of Science ®Google Scholar
• Santos RD, Gidding SS, Hegele RA, et al. Defining severe familial hypercholesterolaemia and the implications for clinical management: a consensus statement from the international atherosclerosis society severe familial hypercholesterolemia panel. Lancet Diabetes Endocrinol. 2016 Oct;4(10):850–861.
   PubMed Web of Science ®Google Scholar
• McGowan MP, Hosseini Dehkordi SH, Moriarty PM, et al. Diagnosis and treatment of heterozygous familial hypercholesterolemia. J Am Heart Assoc. 2019 Dec 17;8(24):e013225.
   PubMed Web of Science ®Google Scholar
• Reiner Ž. Treatment of children with homozygous familial hypercholesterolaemia. Eur J Prev Cardiol. 2018 Jul;25(10):1095–1097.
   PubMed Web of Science ®Google Scholar
• Reiner Ž, Sahebkar A. Treatment of children with heterozygous familial hypercholesterolemia. Int J Cardiol. 2020 Apr;1(304):177–178.
   Google Scholar
• Goldberg AC, Hopkins PN, Toth PP, et al. Familial hypercholesterolemia: screening, diagnosis and management of pediatric and adult patients: clinical guidance from the national lipid association expert panel on familial hypercholesterolemia. J Clin Lipidol. 2011 Jun;5(3 Suppl):S1–8.
   PubMedGoogle Scholar
• Nordestgaard BG, Chapman MJ, Humphries SE, et al. Familial hypercholesterolaemia is underdiagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: consensus statement of the European atherosclerosis society. Eur Heart J. 2013 Dec;34(45):3478–90a.
   PubMed Web of Science ®Google Scholar
• Hovingh GK, Goldberg AC, Moriarty PM. Managing the challenging homozygous familial hypercholesterolemia patient: academic insights and practical approaches for a severe dyslipidemia, a national lipid association masters summit. J Clin Lipidol. 2017 May-Jun;11(3):602–616.
   PubMed Web of Science ®Google Scholar
• Goldstein JL, Brown MS. The LDL receptor. Arterioscler Thromb Vasc Biol. 2009 Apr;29(4):431–438.
   PubMed Web of Science ®Google Scholar
• Berberich AJ, Hegele RA. The complex molecular genetics of familial hypercholesterolaemia. Nat Rev Cardiol. 2019 Jan;16(1):9–20.
   PubMed Web of Science ®Google Scholar
• Garg A, Fazio S, Duell PB, et al. Molecular characterization of familial hypercholesterolemia in a North American Cohort. J Endocr Soc. 2020 Jan 1;4(1):bvz015.
   PubMed Web of Science ®Google Scholar
• Duell PB, Gidding SS, Andersen RL, et al. Longitudinal low density lipoprotein cholesterol goal achievement and cardiovascular outcomes among adult patients with familial hypercholesterolemia: the CASCADE FH registry. Atherosclerosis. 2019 Oct;289:85–93.
   PubMed Web of Science ®Google Scholar
• Vallejo-Vaz AJ, De Marco M, Stevens CAT, et al. Overview of the current status of familial hypercholesterolaemia care in over 60 countries - The EAS familial hypercholesterolaemia studies collaboration (FHSC). Atherosclerosis. 2018 Oct;277:234–255.
   PubMed Web of Science ®Google Scholar
• Williams RR, Hunt SC, Schumacher MC, et al. Diagnosing heterozygous familial hypercholesterolemia using new practical criteria validated by molecular genetics. Am J Cardiol. 1993 Jul 15;72(2):171–176.
   PubMed Web of Science ®Google Scholar
• Risk of fatal coronary heart disease in familial hypercholesterolaemia. Scientific steering committee on behalf of the Simon broome register group. BMJ (Clin Res Ed). 1991 Oct 12;303(6807):893–896.
   PubMedGoogle Scholar
• World Health Organization. Familial hypercholesterolaemia (FH): report of a second WHO consultation; 1998. [cited 2021 Apr 29]. Available from: http://apps.who.int/iris/bitstream/handle/10665/66346/WHO_HGN_FH_CONS_99.2.pdf;jsessionid=961D48D3A63F73082AAC3EB38D01E871?sequence=1
   Google Scholar
• Sturm AC, Knowles JW, Gidding SS, et al. Clinical genetic testing for familial hypercholesterolemia: JACC scientific expert panel. J Am Coll Cardiol. 2018 Aug 7;72(6):662–680.
   PubMed Web of Science ®Google Scholar
• Brown EE, Sturm AC, Cuchel M, et al. Genetic testing in dyslipidemia: a scientific statement from the National Lipid Association. J Clin Lipidol. 2020 Jul-Aug;14(4):398–413.
   PubMed Web of Science ®Google Scholar
• Wiegman A, Hutten BA, de Groot E, et al. Efficacy and safety of statin therapy in children with familial hypercholesterolemia: a randomized controlled trial. Jama. 2004 Jul 21;292(3):331–337.
   PubMed Web of Science ®Google Scholar
• Kusters DM, Wiegman A, Kastelein JJ, et al. Carotid intima-media thickness in children with familial hypercholesterolemia. Circ Res. 2014 Jan 17;114(2):307–310.
   PubMed Web of Science ®Google Scholar
• Kusters DM, Avis HJ, de Groot E, et al. Ten-year follow-up after initiation of statin therapy in children with familial hypercholesterolemia. Jama. 2014 Sep 10;312(10):1055–1057.
   PubMed Web of Science ®Google Scholar
• Vuorio A, Kuoppala J, Kovanen PT, et al. Statins for children with familial hypercholesterolemia. Cochrane Database Syst Rev. 2017 7;7;Jul(7):Cd006401.
   Google Scholar
• Braamskamp M, Langslet G, McCrindle BW, et al. Effect of rosuvastatin on carotid intima-media thickness in children with heterozygous familial hypercholesterolemia: the CHARON study (hypercholesterolemia in children and adolescents taking rosuvastatin open label). Circulation. 2017 Jul 25;136(4):359–366.
   PubMed Web of Science ®Google Scholar
• Luirink IK, Wiegman A, Kusters DM, et al. 20-year follow-up of statins in children with familial hypercholesterolemia. N Engl J Med. 2019 Oct 17;381(16):1547–1556.
   PubMed Web of Science ®Google Scholar
• Mach F, Baigent C, Catapano AL, et al. 2019 ESC/EAS guidelines for the management of dyslipidaemias: lipid modification to reduce cardiovascular risk. Eur Heart J. 2020 Jan 1;41(1):111–188.
   PubMed Web of Science ®Google Scholar
• Cuchel M, Bruckert E, Ginsberg HN, et al. Homozygous familial hypercholesterolaemia: new insights and guidance for clinicians to improve detection and clinical management. A position paper from the consensus panel on familial hypercholesterolaemia of the European atherosclerosis society. Eur Heart J. 2014 Aug 21;35(32):2146–2157.
   PubMed Web of Science ®Google Scholar
• Jacobson TA, Ito MK, Maki KC, et al. National lipid association recommendations for patient-centered management of dyslipidemia: part 1--full report. J Clin Lipidol. 2015 Mar-Apr;9(2):129–169.
   PubMed Web of Science ®Google Scholar
• Grundy SM, Stone NJ, Bailey AL, et al. AHA/ACC/AACVPR/AAPA/ABC/ACPM/ADA/AGS/APhA/ASPC/NLA/PCNA guideline on the management of blood cholesterol: a report of the American college of cardiology/American Heart association task force on clinical practice guidelines. J Am Coll Cardiol. 2019;73(24):e285-e350.
   Web of Science ®Google Scholar
• Handelsman Y, Jellinger PS, Guerin CK, et al. Consensus statement by the American association of clinical endocrinologists and American college of endocrinology on the management of dyslipidemia and prevention of cardiovascular disease algorithm - 2020 executive summary. Endocr Pract. 2020 Oct;26(10):1196–1224.
   PubMed Web of Science ®Google Scholar
• Rallidis LS, Liberopoulos EN, Vlachopoulos C, et al. Very high-risk familial hypercholesterolaemia patients in real life: the remaining gap in achieving the current LDL-C targets despite the use of PCSK9 inhibitors. Atherosclerosis. 2020 Sep;309:67–69.
   PubMed Web of Science ®Google Scholar
• Wong ND, Young D, Zhao Y, et al. Prevalence of the American college of cardiology/American heart association statin eligibility groups, statin use, and low-density lipoprotein cholesterol control in US adults using the National Health and nutrition examination survey 2011-2012. J Clin Lipidol. 2016 Sep-Oct;10(5):1109–1118.
   PubMed Web of Science ®Google Scholar
• Rached F, Santos RD. Beyond statins and PCSK9 inhibitors: updates in management of familial and refractory hypercholesterolemias. Curr Cardiol Rep. 2021 Jun 3;23(7):83.
   PubMed Web of Science ®Google Scholar
• Ray KK, Landmesser U, Leiter LA, et al. Inclisiran in patients at high cardiovascular risk with elevated LDL cholesterol. N Engl J Med. 2017 Apr 13;376(15):1430–1440.
   PubMed Web of Science ®Google Scholar
• Raal FJ, Kallend D, Ray KK, et al. Inclisiran for the treatment of heterozygous familial hypercholesterolemia. N Engl J Med. 2020 Apr 16;382(16):1520–1530.
   PubMed Web of Science ®Google Scholar
• Ray KK, Wright RS, Kallend D, et al. Two phase 3 trials of inclisiran in patients with elevated LDL cholesterol. N Engl J Med. 2020 Apr 16;382(16):1507–1519.
   PubMed Web of Science ®Google Scholar
• Hovingh GK, Lepor NE, Kallend D, et al. Inclisiran durably lowers low-density lipoprotein cholesterol and proprotein convertase subtilisin/kexin type 9 expression in homozygous familial hypercholesterolemia: the ORION-2 pilot study. Circulation. 2020 Jun 2;141(22):1829–1831.
   PubMed Web of Science ®Google Scholar
• Santos RD, Stein EA, Hovingh GK, et al. Long-term evolocumab in patients with familial hypercholesterolemia. J Am Coll Cardiol. 2020 Feb 18;75(6):565–574.
   PubMed Web of Science ®Google Scholar
• Morelli MB, Chavez C, Santulli G. Angiopoietin-like proteins as therapeutic targets for cardiovascular disease: focus on lipid disorders. Expert Opin Ther Targets. 2020 Jan;24(1):79–88.
   PubMed Web of Science ®Google Scholar
• Ruscica M, Zimetti F, Adorni MP, et al. Pharmacological aspects of ANGPTL3 and ANGPTL4 inhibitors: new therapeutic approaches for the treatment of atherogenic dyslipidemia. Pharmacol Res. 2020 Mar;153:104653.
   PubMed Web of Science ®Google Scholar
• Ling P, Zheng X, Luo S, et al. Targeting angiopoietin-like 3 in atherosclerosis: from bench to bedside. Diabetes Obes Metab. 2021 May 28. DOI:10.1111/dom.14450. .
   Web of Science ®Google Scholar
• Warden BA, Duell PB. Evinacumab: anti-ANGPTL3 (angiopoietin-like protein 3) monoclonal antibody Treatment of homozygous familial hypercholesterolemia Treatment of dyslipidemia and cardiovascular disease. Drugs Future. 2020;45(9):619–631.
   Web of Science ®Google Scholar
• Stitziel NO, Khera AV, Wang X, et al. ANGPTL3 deficiency and protection against coronary artery disease. J Am Coll Cardiol. 2017 Apr 25;69(16):2054–2063.
   PubMed Web of Science ®Google Scholar
• Minicocci I, Montali A, Robciuc MR, et al. Mutations in the ANGPTL3 gene and familial combined hypolipidemia: a clinical and biochemical characterization. J Clin Endocrinol Metab. 2012 Jul;97(7):E1266–75.
   PubMed Web of Science ®Google Scholar
• Dewey FE, Gusarova V, Dunbar RL, et al. Genetic and Pharmacologic Inactivation of ANGPTL3 and cardiovascular disease. N Engl J Med. 2017 Jul 20;377(3):211–221.
   PubMed Web of Science ®Google Scholar
• Ando Y, Shimizugawa T, Takeshita S, et al. A decreased expression of angiopoietin-like 3 is protective against atherosclerosis in apoE-deficient mice. J Lipid Res. 2003 Jun;44(6):1216–1223.
   PubMed Web of Science ®Google Scholar
• Graham MJ, Lee RG, Brandt TA, et al. Cardiovascular and metabolic effects of ANGPTL3 antisense oligonucleotides. N Engl J Med. 2017 Jul 20;377(3):222–232.
   PubMed Web of Science ®Google Scholar
• U.S.food and drug administration center for drug evaluation and research – summary review. Evkeeza (evinacumab-dgnb). cited 2021 May 5. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761181Orig1s000SumR.pdf
   Google Scholar
• Takiguchi S, Ayaori M, Yakushiji E, et al. Hepatic overexpression of endothelial lipase lowers high-density lipoprotein but maintains reverse cholesterol transport in mice: role of scavenger receptor class B Type I/ATP-binding cassette transporter A1-dependent pathways. Arterioscler Thromb Vasc Biol. 2018 Jul;38(7):1454–1467.
   PubMed Web of Science ®Google Scholar
• Gusarova V, Alexa CA, Wang Y, et al. ANGPTL3 blockade with a human monoclonal antibody reduces plasma lipids in dyslipidemic mice and monkeys. J Lipid Res. 2015 Jul;56(7):1308–1317.
   PubMed Web of Science ®Google Scholar
• Wang Y, Gusarova V, Banfi S, et al. Inactivation of ANGPTL3 reduces hepatic VLDL-triglyceride secretion. J Lipid Res. 2015 Jul;56(7):1296–1307.
   PubMed Web of Science ®Google Scholar
• Pouwer MG, Pieterman EJ, Worms N, et al. Alirocumab, evinacumab, and atorvastatin triple therapy regresses plaque lesions and improves lesion composition in mice. J Lipid Res. 2020 Mar;61(3):365–375.
   PubMed Web of Science ®Google Scholar
• Prescribing information. Evkeeza (evinacumab). Tarrytown,Ny: Regeneron Pharmaceuticals, LLC; 2021.
   Google Scholar
• Prescribing Information. Praluent (alirocumab). Bridgewater, NJ: Sanofi-Aventis U.S., LLC; 2019.
   Google Scholar
• Ahmad Z, Banerjee P, Hamon S, et al. Inhibition of angiopoietin-like protein 3 with a monoclonal antibody reduces triglycerides in hypertriglyceridemia. Circulation. 2019 Aug 6;140(6):470–486.
   PubMed Web of Science ®Google Scholar
• Gaudet D, Gipe DA, Pordy R, et al. ANGPTL3 inhibition in homozygous familial hypercholesterolemia. N Engl J Med. 2017 Jul 20;377(3):296–297.
   PubMed Web of Science ®Google Scholar
• Raal FJ, Rosenson RS, Reeskamp LF, et al. Evinacumab for homozygous familial hypercholesterolemia. N Engl J Med. 2020 Aug 20;383(8):711–720.
   PubMed Web of Science ®Google Scholar
• Reeskamp LF, Nurmohamed NS, Bom MJ, et al. Marked plaque regression in homozygous familial hypercholesterolemia. Atherosclerosis. 2021 Jun;327:13–17.
   PubMed Web of Science ®Google Scholar
• Rosenson RS, Burgess LJ, Ebenbichler CF, et al. Evinacumab in patients with refractory hypercholesterolemia. N Engl J Med. 2020 Dec 10;383(24):2307–2319.
   PubMed Web of Science ®Google Scholar
• ClinicalTrials.gov [internet]. Bethesda (MD): National Library of Medicine (US). cited 2020 Apr 30. Available from https://clinicaltrials.gov/ct2/results?cond=evinacumab&term=&cntry=&state=&city=&dist=
   Google Scholar
• U.S.food and drug administration center for drug evaluation and research – risk assessment and risk mitigation review(s). Evkeeza (evinacumab-dgnb). [ cited 2021 May 5]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2021/761181Orig1s000RiskR.pdf
   Google Scholar
• Evinacumab-dgnb (Evkeeza; Regeneron Pharmaceuticals, Inc.) is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism. Pharmacy Times. [cited 2021 May 12]. Available from: https://www.pharmacytimes.com/view/fda-approves-first-in-class-therapy-for-ultra-rare-form-of-high-cholesterol
   Google Scholar
• Nicholls SJ, Puri R, Anderson T, et al. Effect of evolocumab on progression of coronary disease in statin-treated patients: the GLAGOV randomized clinical trial. Jama. 2016 Dec 13;316(22):2373–2384.
   PubMed Web of Science ®Google Scholar
• Baigent C, Keech A, Kearney PM, et al. Efficacy and safety of cholesterol-lowering treatment: prospective meta-analysis of data from 90,056 participants in 14 randomised trials of statins. Lancet. 2005 Oct 8;366(9493):1267–1278.
   PubMed Web of Science ®Google Scholar
• Warden BA, Duell PB. Volanesorsen for treatment of patients with familial chylomicronemia syndrome. Drugs Today (Barc). 2018 Dec;54(12):721–735.
   PubMedGoogle Scholar
• Rosenson RS. A phase 2 trial of the efficacy and safety of evinacumab in patients with severe hypertriglyceridemia. Abstract 406-19, ACC 2021 Scientific Session, 15-17 May.
   Google Scholar
• National Cholesterol Education Program (NCEP) Expert Panel on Detection, Evaluation, and Treatment of High Blood Cholesterol in Adults (Adult Treatment Panel III). Third report of the national cholesterol education program (NCEP) expert panel on detection, evaluation, and treatment of high blood cholesterol in adults (adult treatment panel III) final report. Circulation. 2002 Dec 17;106(25):3143–3421.
   PubMed Web of Science ®Google Scholar
• Voight BF, Peloso GM, Orho-Melander M, et al. Plasma HDL cholesterol and risk of myocardial infarction: a mendelian randomisation study. Lancet. 2012 Aug 11;380(9841):572–580.
   PubMed Web of Science ®Google Scholar
• Holmes MV, Asselbergs FW, Palmer TM, et al. Mendelian randomization of blood lipids for coronary heart disease. Eur Heart J. 2015 Mar 1;36(9):539–550.
   PubMed Web of Science ®Google Scholar