52
Views
7
CrossRef citations to date
0
Altmetric
Review

Development of proprotein convertase subtilisin/kexin type 9 inhibitors and the clinical potential of monoclonal antibodies in the management of lipid disorders

Pages 421-433 | Published online: 10 Nov 2016

References

  • Expert Panel on Detection, Evaluation and Treatment of High Blood Cholesterol in AdultsExecutive summary of the third report of the national Cholesterol Education Programme (NCEP). Expert Panel on Detection, Evaluation and Treatment of High Blood Cholesterol in Adults (Adult Treatment Panel III)JAMA2001285192486249711368702
  • O’KeefeJHJrCordainLHarrisWHMoeRMVogelROptimal low density lipoprotein is 50–70 mg/dl; lower the better and physiologically normalJ Am Coll Cardiol200443112142214615172426
  • AhmedSCannonCPMurphySABraunwaldEAcute coronary syndrome and diabetes: is intensive lipid lowering beneficial? Result of the PROVE IT-TIMI 22 trialEur Heart J200627192323232916954134
  • Cholesterol Treatment Trialists’ (CTT) CollaborationBaigentCBlackwellLEmbersonJEfficacy and safety of more intensive lowering of LDL cholesterol: a meta analysis of data from 170,000 participants in 26 randomized trialsLancet201037697531670168121067804
  • StoneNJRobinsonJGLichtensteinAHAmerican College of Cardiology/American Heart Association Task Force on Practice Guidelines. 2013 ACC/AHA guideline on the treatment of blood cholesterol to reduce atherosclerotic cardiovascular risk in adults: a report of the American College of Cardiology/American Heart Association Task Force on Practice GuidelinesJ Am Coll Cardiol20146325 Pt B2889293424239923
  • PerkJDe BackerGGohlkeHEuropean Association for Cardiovascular Prevention & Rehabilitation (EACPR); ESC Committee for Practice Guidelines (CPG). European guidelines on cardiovascular disease prevention in clinical practice (version 2012). The Fifth Joint Task Force of the European Society of Cardiology and Other Societies on Cardiovascular Disease Prevention in Clinical Practice (constituted by representatives of nine societies and by invited experts)Eur Heart J201233131635170122555213
  • AndersonTJGreogoireJHegeleRA2012 update of the Canadian Cardiovascular Society guidelines for the diagnosis and treatment of dyslipidemia for the prevention of cardiovascular disease in adultsCan J Cardiol201329215116723351925
  • BoekholdtSMHovinghGKMoraSVery low levels of atherogenic lipoproteins and risk for cardiovascular events: a meta-analysis of statin trialsJ Am Coll Cardiol201464548549425082583
  • CannonCPBlazingMAGiuglianoRPIMPROVE-IT InvestigatorsEzetimibe added to statin therapy after acute coronary syndromes. IMPROVED reduction of outcome Vytorin Efficacy International Trial (IMPROVE-IT)N Engl J Med20153722387239726039521
  • SeidahNGProprotein convertase subtilisin kexin 9 (PCSK9) inhibitors in the treatment of hypercholesterolemia and other pathologiesCurr Pharm Des201319173161317223317404
  • EverettBMSmithRJHiattWRReducing LDL with PCSK9 inhibitors – the clinical benefit of lipid drugsN Engl J Med2015373171588159126444323
  • StoekebroekRMKasteleinJJHuijgenRPCSK9 inhibition: the way forward in the treatment of dyslipidemiaBMC Med20151325826326456772
  • GiuglianoRPSabatineMSAre PCKS9 inhibitors the next breakthrough in the cardiovascular field?J Am Coll Cardiol201565242638265126088304
  • AbifadelMVarretMRabesJPMutations in PCSK9 cause autosomal dominant hypercholesterolemiaNat Genet200334215415612730697
  • SeidahNGBenjannetSWickhamLThe secretory proprotein convertase neural apoptosis-regulated convertase I (NARC-1): liver regeneration and neuronal differentiationProc Natl Acad Sci U S A2003100392893312552133
  • CohenJCBoerwinkleEMosleyTHJrHobbsHHSequence variations in PCSK9, low LDL, and protection against coronary heart diseaseN Engl J Med20063541212647216554528
  • LambertGPetridesFChateliasMElevated plasma PCSK9 levels are equally detrimental for patients with non familial hypercholesterolemia and heterozygous familial hypercholesterolemia, irrespective of low density lipoprotein receptor defectsJ Am Coll Cardiol2014632223657524632287
  • HoriMIshiharaMYuasaYRemoval of plasma mature and furin-cleaved proprotein convertase subtilisin/kexin 9 by low density lipoprotein-apheresis in familial hypercholesterolemia: development and application of a new assay for PCSK9J Clin Endocrinol Metab20151001E41E4925313916
  • TavoriHChristianDMinnierJPCSK9 association with lipoprotein (a)Circ Res2016119293527121620
  • GiuglianoRPDesaiNRKohliPLAPLACE-TIMI 57 InvestigatorsEfficacy, safety and tolerability of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 in combination with a statin in patients with hypercholesterolemia (LAPLACE-TIMI 57): a randomised, placebo-controlled, dose-ranging, phase 2 studyLancet201238095982007201723141813
  • KorenMJScottRKimJBEfficacy, safety and tolerability of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 as monotherapy in patients with hypercholesterolemia (MENDEL): a randomised, double-blind, placebo-controlled, phase 2 studyLancet201238098581995200623141812
  • SullivanDOlssonAGScottREffect of a monoclonal antibody to PCSK9 on low-density lipoprotein cholesterol levels in statin-intolerant patients: the GAUSS randomized trialJAMA2012308232497250623128163
  • SteinEAHonarpourNWassermanSMXuFScottRRaalFJEffect of the proprotein convertase subtilisin/kexin 9 monoclonal antibody, AMG 145, in homozygous familial hypercholesterolemiaCirculation2013128192113212024014831
  • RaalFScottRSomaratneRLow-density lipoprotein cholesterol-lowering effects of AMG 145, a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease in patients with heterozygous familial hypercholesterolemia: the Reduction of LDL-C with PCSK9 inhibition in Heterozygous Familial Hypercholesterolemia Disorder (RUTHERFORD) randomised trialCirculation2012126202408241723129602
  • RothEMMcKenneyJMHanotinCAssetGSteinEAAtorvastatin with or without an antibody to PCSK9 in primary hypercholesterolemiaN Engl J Med201236720189190023113833
  • SteinEAGipeDBergeronJEffect of a monoclonal antibody to PCSK9, alirocumab to reduce low-density lipoprotein cholesterol in patients with heterozygous familial hypercholesterolemia on stable statin dose with or without ezetimibe therapy: a phase 2 randomised controlled trialLancet20123809836293622633824
  • McKenneyJMKorenMJKereiakesDJHanotinCFerrandACSteinEASafety and efficacy of a monoclonal antibody to proprotein convertase subtilisin/kexin type 9 serine protease, alirocumab, in patients with primary hypercholesterolemia receiving ongoing stable atorvastatin therapyJ Am Coll Cardiol201259252344235322463922
  • BallantyneCMNeutelJCroppAResults of Bococizumab, a monoclonal antibody against proprotein convertase subtilisin/kexin type 9, from a randomized, placebo-controlled, dose-ranging study in statin-treated subjects with hypercholesterolemiaAm J Cardiol201511591212122125784512
  • KorenMJLundqvistPBologneseMMENDEL-2 InvestigatorsAnti-PCSK9 monotherapy for hypercholesterolemia: the MENDEL-2 randomized, controlled phase III clinical trial of evolocumabJ Am Coll Cardiol201463232531254024691094
  • StroesEColquhounDSullivanDGAUSS-2 InvestigatorsAnti-PCSK9 antibody effectively lowers cholesterol in patients with statin intolerance: the GAUSS-2 randomized, placebo-controlled phase 3 clinical trial of evolocumabJ Am Coll Cardiol201463232541254824694531
  • KorenMJGiuglianoRPRaalFJOSLER InvestigatorsEfficacy and safety of longer-term administration of evolocumab in patients with hypercholesterolemia: 52-week results from the Open-Label Study of Long-Term Evaluation against LDL-C (OSLER) randomized trialCirculation2014129223424324255061
  • RaalFJHonarpourNBlomDJTESLA InvestigatorsInhibition of PCSK9 with evolocumab in homozygous familial hypercholesterolemia (TESLA Part B): a randomized, double-blind, placebo-controlled trialLancet2015385996534135025282520
  • RobinsonJGNedergaardBSRogersWJLAPLACE-2 InvestigatorsEffect of evolocumab or ezetimibe added to moderate – or high-intensity statin therapy on LDLc lowering in patients with hypercholesterolemia: the LAPLACE-2 randomized clinical trialJAMA2014311181870188224825642
  • BloomDJHalaTBologneseMA 52-week placebo-controlled trial of evolocumab in hyperlipidemiaN Engl J Med2014370191809181924678979
  • RaalFJSteinEADufourRRUTHERFORD-2 InvestigatorsPCSK9 inhibition with evolocumab (AMG145) in heterozygous familial hypercholesterolemia (RUTHERFORD-2): a randomised, double-blind, placebo-controlled trialLancet2015385996533134025282519
  • RobinsonJGFarnierMKrempfMEfficacy and safety of alirocumab in reducing lipids and cardiovascular events (ODYSSEY LONG TERM Investigators)N Engl J Med2015372161489149925773378
  • CannonCPCariouBBlomDODYSSEY COMBO II InvestigatorsEfficacy and safety of alirocumab in high cardiovascular risk patients with inadequately controlled hypercholesterolemia on maximally tolerated doses of statins: the ODYSSEY COMBO II randomised controlled trialEur Heart J201536191186119425687353
  • KasteleinJJRobinsonJGFarnierMEfficacy and safety of alirocumab in patients with heterozygous familial hypercholesterolemia not adequately controlled with current lipid-lowering therapy: design and rationale of the ODYSSEY FH studiesCardiovasc Drugs Ther201428328128924842558
  • MoriartyPMJacobsonTABruckertEEfficacy and safety of alirocumab, a monoclonal antibody to PCSK9, in statin-intolerant patients: design and rationale of ODYSSEY ALTERNATIVE, a randomised phase 3 trialJ Clin Lipidol20148655456125499937
  • RothEMTaskinenMRGinsbergHNMonotherapy with the PCSK9 inhibitor alirocumab versus ezetimibe in patients with hypercholesterolemia: results of a 24 week, double-blind, randomized Phase 3 trialInt J Cardiol20141761556125037695
  • SabatineMSGiuglianoRPWiviottSDEfficacy and safety of Evolocumab in reducing lipids and cardiovascular events (Open-Label Study of Long-term Evaluation against LDL Cholesterol Investigators) (OSLER)N Engl J Med2015372161500150925773607
  • ZhangXLZhuQQZhuLSafety and efficacy of anti-PCSK9 antibodies: a meta-analysis of 25 randomized, controlled trialsBMC Med20151312326099511
  • NavareseEPKolodziejczakMSchulzeVEffects of proprotein convertase subtilsin/kexin type 9 antibodies in adults with hypercholesterolemia: a systematic review and meta-analysisAnn Intern Med201516314149
  • LipinskiMJBenedettoUEscarcegaROThe impact of proprotein convertase subtilisin/kexin type 9 serine protease inhibitor on lipids levels and outcomes in patients with primary hypercholesterolemia: a network meta-analysisEur Heart J201637653654526578202
  • ICER Draft Report 2015. PCSK9 inhibitors for treatment of high cholesterol: effectiveness, value and value-based price benchmarksInstitute for Clinical and Economic Review Published online 8 September 2015
  • NordestgaardBGChapmanMJHumphriesSEEuropean Atherosclerosis Society Consensus Panel. Familial hypercholesterolemia is under diagnosed and undertreated in the general population: guidance for clinicians to prevent coronary heart disease: consensus statement of the European Atherosclerosis SocietyEur Heart J2013344534783490a23956253
  • BennMWattsGFTybjaerg-HansenANordestgaardBGMutations causative of familial hypercholesterolemia: screening of 98098 individuals from the Copenhagen General Population Study estimated a prevalence of 1 in 217Eur Heart J201637171384139426908947
  • WiegmanAGiddingSSWattsGFEuropean Atherosclerosis Society Consensus Panel. Familial hypercholesterolemia in children and adolescents: gaining decades of life by optimizing detection and treatmentEur Heart J201536362425243726009596
  • VuorioAWattsGFKovenanPTInitiation of PCSK9 inhibition in patients with heterozygous familial hypercholesterolemia entering adulthood: a new design for living with a high-risk condition?Eur Heart J2016371353135626851704
  • RidkerPMMoraSRoseLJUPITER Trial Study Group. Percent reduction in LDL cholesterol following high-intensity statin therapy: potential implications for guidelines and for the prescription of emerging lipid-lowering agentsEur Heart J201637171373137926916794
  • GlueckCJShahPGoldenbergNEligibility for PCSK9 treatment in 734 hypercholesterolemic patients referred to a regional cholesterol treatment centre with LDL cholesterol ≥ 70 mg/dl despite maximal tolerated cholesterol lowering therapyLipids Health Dis2016155526968977
  • TorjesenINICE draft guidance recommends PCSK9 inhibitors for high risk cardiovascular patientsPharm J2016296788920201125
  • KaziDSMoranAECoxsonPGCost-effectiveness of PCSK9 inhibitor therapy in patients with homozygous familial hypercholesterolemia or atherosclerotic cardiovascular diseaseJAMA2016316774375327533159
  • WintraubWSDanielsSRBurekLEAmerican Heart Association Advocacy Coordinating Committee; Council on Cardiovascular Disease in the Young; Council on the Kidney in Cardiovascular Disease; Council on Epidemiology and Prevention; Council on Cardiovascular Nursing; Council on Arteriosclerosis; Thrombosis and Vascular Biology; Council on Clinical Cardiology, and Stroke Council. Value of primordial and primary prevention for cardiovascular disease: a policy statement from American Heart AssociationCirculation2011124896799021788592
  • GandraSRVillaGFonarowGCCost-effectiveness of LDLc lowering with evolocumab in patients with high cardiovascular risk in the United StatesClin Cardiol201639631332027092712
  • BarterPJCaulfiedMErikssonMEffects of Torcetrapib in patients at high risk for coronary eventsN Engl J Med20073572109212217984165
  • HPS-2 THRIVE Trial Collaborative GroupLandrayMJHaynesRHopewellJCEffects of extended release niacin with laropiprant in high risk patientsN Engl J Med2014371320321225014686