全球首个糖尿病性视网膜病变（DR）药物诞生，FDA批准罗氏Lucentis第四个适应症

2015年2月12日讯/生物谷BIOON/--瑞士制药巨头罗氏（Roche）近日宣布，FDA已批准眼科药物Lucentis（ranibizumab，雷珠单抗）用于糖尿病性黄斑水肿（DME）患者糖尿病性视网膜病变（DR）的治疗。此次批准，标志着Lucentis成为全球首个糖尿病性视网膜病变（DR）治疗药物。此前，FDA已授予Lucentis突破性疗法认定和优先审查资格。
Lucentis于2006年上市，已获FDA批准的3个适应症分别为：糖尿病性黄斑水肿（DME，2006年）、视网膜静脉阻塞继发黄斑水肿（RVO-ME，2010年）和湿性年龄相关性黄斑变性（wet-AMD，2012）。此次糖尿病性视网膜病变（DR），标志着Lucentis在美国的第四个适应症，在相关临床试验中，Lucentis不仅显著改善了糖尿病性黄斑水肿（DME）患者的视力，同时也使糖尿病性视网膜病变（DR）的损伤程度得到了临床意义的显著改善。
目前，在眼科治疗领域，拜耳眼科药物Eylea正与罗氏Lucentis展开着激烈的竞争。Eylea于2011年底上市，尽管晚了Lucentis几年，但该药上市以来发展势头迅猛，适应症个数及全球销售一再刷新，并连续数次超过业界预期，在相关疾病领域已对罗氏Lucentis形成了严峻的挑战，包括视网膜静脉阻塞继发黄斑水肿（RVO-ME）和糖尿病性黄斑水肿（DME）。去年10月，在一项独立疗效比较研究中，治疗糖尿病性黄斑水肿（DME）时，Eylea疗效击败Lucentis和Avastin，该项研究使得Eylea在DME领域更具影响力。
此次，Lucentis率先拿下糖尿病性视网膜病变（DR）适应症，也标志着对Eylea强有力的反击。不过，Eylea也在步步逼近，FDA目前正在审查Eylea的DR适应症申请，该药于去年9月和12月也收获了FDA的突破性疗法认定和优先审查资格。这也意味着罗氏万不可掉以轻心，须抓住首发优势，竭尽所能在糖尿病性视网膜病变（DR）领域快速抢占市场。
另一方面，当前肿瘤治疗领域竞争异常激烈，尤其是PD-1/PD-L1抑制剂和CAR-T细胞疗法在相关临床取得巨大成功的背景下，作为肿瘤领域的巨头，罗氏面对的挑战可想而知。业界认为，Lucentis作为罗氏最成功的非肿瘤类药物，将在接下来的2015年，肩负更重要的使命。
关于Lucentis：
Lucentis是一种人源化的治疗性抗体片段，旨在阻断所有生物活性形式的血管内皮细胞生长因子A（VEGF-A），该因子的水平在湿性AMD和其他眼科疾病（如糖尿病性黄斑水肿（DME）、视网膜静脉阻塞（RVO））升高。Lucentis于2006年上市，由罗氏（Roche）旗下基因泰克（Genentech）和诺华合作开发，罗氏拥有Lucentis在美国的商业化权利，诺华则拥有该药在美国以外国家和地区的独家权利。
英文原文：FDAapprovesRoche’sLucentis(ranibizumabinjection)fortreatmentofdiabeticretinopathyinpeoplewithdiabeticmacularedema
-Firsteyemedicineapprovedfortreatmentofdiabeticretinopathywithdiabeticmacularedema
-GrantedBreakthroughTherapyDesignationandPriorityReviewbyFDA
-Diabeticmacularedemacanoccuratanystageofdiabeticretinopathy,aleadingcauseofblindnessinAmericanadults.1
-FourthLucentisindicationfortreatmentofseriouseyediseasessince2006
Roche(SIX:RO,ROG;OTCQX:RHHBY)announcedtodaythattheU.S.FoodandDrugAdministration(FDA)approvedLucentis?(ranibizumabinjection)forthetreatmentofdiabeticretinopathy(DR),inpeoplewithdiabeticmacularedema(DME).DMEimpactsnearly750,000Americans,about10percentofpeoplewithDR.
TheFDAgrantedLucentisBreakthroughTherapyDesignationandPriorityReviewforthisindicationbasedonresultsfromtheRISEandRIDEPhaseIIIclinicaltrials.
“Whiletherearevariousoptionsfortreatingdiabeticmacularedema,beforetodaynonewereapprovedshowingimprovementinretinopathy,”saidSandraHorning,M.D.,chiefmedicalofficerandheadofGlobalProductDevelopment.“Withtodaysapproval,peoplewithdiabeticmacularedemanowhaveaFDA-approvedmedicinethatshowedmeaningfulimprovementsinretinaldamagefromdiabetes,inadditiontotheestablishedimprovementinvision.”
Almost29millionAmericanshavediabetes.2Thelongerpeoplehavediabetes,especiallyifitispoorlymanaged,thehighertheirriskfordevelopingDR.Itiscausedbyelevatedbloodsugarlevelsdamagingthefinebloodvesselsoftheretina,thelight-sensitivetissueatthebackoftheeyenecessaryforgoodvision.
DRwithDMEisacommondiabeticeyediseaseandaleadingcauseofblindnessinAmericanadultsunder55.1DRwithDMEcanleadtoconditionsthatthreatenvision.
TheFDAdesignatesBreakthroughTherapytoamedicineifitisintendedtotreataseriousorlife-threateningdiseaseandifpreliminaryclinicalresearchsuggestsitmayprovidesubstantialimprovementonclinicallysignificantendpointsoverexistingtherapies.
TheFDAgrantsPriorityReviewtomedicinesthat,ifapproved,wouldhavethepotentialtoprovidesignificantimprovementsinthesafetyoreffectivenessofthetreatment,diagnosis,orpreventionofseriousconditionswhencomparedtostandardapplications.
In2012,LucentiswasthefirstmedicineapprovedbytheFDAforthetreatmentofDME.Lucentishasalsobeenanimportantoptionforpatientswithwetage-relatedmaculardegeneration(wetAMD)since2006andmacularedemafollowingretinalveinocclusion(RVO)since2010.
AboutRISEandRIDE
RISEandRIDEaretwoidentically-designed,parallel,double-masked,shamtreatment-controlledtrialsin759patientswithDRandDMEatbaselinewhowererandomizedintothreegroupstoreceivemonthlytreatmentwith0.3mgLucentis,0.5mgLucentisorshaminjection.TheprimaryoutcomeinRISEandRIDEwasvisualacuitygainat24monthsforDMEpatients.
ThesafetyandefficacyofLucentisforthetreatmentofDRwithDMEwasassessedoverthreeyearsinpatientswithbaselineDRseverityscoresrangingfrom10to75inthestudyeye(ontheETDRSdiabeticretinopathyseverityscale).Secondaryandexploratoryoutcomeswereevaluatedat24months.AtMonth24,ahigherproportionofpatientshadobservedathree-steporbetterimprovementoftheirdiseasecomparedtosham,asdeterminedbycolorfundusphotography.ThesafetyintheRISEandRIDEPhaseIIItrialswasconsistentwithpreviousstudies.
Inthethirdyearofthestudies,patientsfromthecontrolgrouphadtheoptiontocrossovertoreceivemonthlytreatmentwith0.5mgLucentis;patientsoriginallyrandomizedto0.3mgor0.5mgLucentiscontinuedtoreceivethesamedoseandallpatientswerefollowedfor12additionalmonths.The0.3mgdoseofLucentisisapprovedforbothDMEandforDRinpeoplewithdiabeticmacularedema.
AboutLucentis
Lucentisisavascularendothelialgrowthfactor(VEGF)inhibitordesignedtobindtoandinhibitVEGF-A,aproteinthatisbelievedtoplayacriticalroleintheformationofnewbloodvessels(angiogenesis)andthehyperpermeability(leakiness)ofthevessels.
LucentisisFDA-approvedforthetreatmentofwetAMD,macularedemafollowingRVO,DMEandDRinpeoplewithDME.GenentechhasconductedeightkeyclinicaltrialswithLucentis.Themedicinehasbeenstudiedin21clinicaltrialsworldwideinmorethan9,080patients.
LucentiswasdevelopedbyGenentech.ThecompanyretainscommercialrightsintheU.S.andNovartishasexclusivecommercialrightsfortherestoftheworld.
OutsidetheU.S.,Lucentisisapprovedinmorethan100countriestotreatpatientswithwetAMD,forthetreatmentofDME,andduetomacularedemasecondarytobothbranchretinalveinocclusion(BRVO)andcentralretinalveinocclusion(CRVO).
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