Actelion公司肺动脉高压药物Opsumit获日本批准

2015年4月1日讯/生物谷BIOON/--Actelion制药公司肺动脉高压药物Opsumit（macitentan）近日在监管方面收获第3个重大里程碑，该药近期获日本批准，作为一种每日一次的口服药物，用于肺动脉高压（PAH）的治疗，以延缓病情进展。此前，Opsumit已于2013年底接连获批在美国和欧盟2大主要市场上市。
Opsumit是Actelion公司另一款PAH口服药物Tracleer的继任者，后者是该公司最畅销的产品，2012年销售额高达15亿瑞士法郎。然而，Tracleer将于2015年面临专利悬崖，该公司正指望Opsumit来弥补Tracleer专利到期所致的销售损失，该药将与市面上的其他PAH药物竞争，包括吉利德的Letairis、拜耳的Adempas（riociguat）、辉瑞的万艾可（Vigra，即伟哥）、礼来的希爱力（Cialis）及相关仿制药。
Opsumit是一种双效内皮素受体拮抗剂（ERA），能够松弛肺动脉并降低血压，该药与同类其他药物一样都具有一个黑框警示，禁止用于孕妇，因为这类药物可能对胎儿造成伤害。Opsumit的安全性和疗效已在迄今规模最大、开展时间最长的PAH临床试验SERAPHIN中得到证实，有近40个国家151个中心参与了研究。数据显示，与安慰剂相比，10mg剂量Opsumit使发病/死亡风险显著降低45%，同时使PAH相关住院或死亡风险显著降低50%。
肺动脉高压（PAH）是一种罕见病，全球发病率仅为百万分之十五至五十，特征是肺动脉血压异常升高，该病是一种慢性、极度严重、致死性疾病。尽管PAH罕见，但在高危群体中发病率显著升高，如HIV感染者中发病率约为0.5%，镰状细胞病患者中发病率约2-3.75%，而系统性硬化症患者中的发病率高达7-12%。
英文原文：ActelionLtd’sPAHDrugOpsumitGetsTheGreenLightInJapan
ActelionLtd’sdrugOpsumitwasgrantedapprovalyesterdaybytheJapaneseMinistryofHealth,LaborandWelfare,forthetreatmentofpulmonaryarterialhypertension
ActelionLtd(OTCMKTS:ALIOF)’spulmonaryarterialhypertensiondrug,Opsumit,haswonfinalapprovalfromtheJapaneseMinistryofHealth,LaborandWelfare.Thedecision,announcedonThursday,meansthedrug,alsoknownbyitschemicalnamemacitentancanbelaunchedimmediatelyontothelocalmarket.
Opsumit,,willbeco-marketedinJapanbyActelionPharmaceuticalsJapanandNIPPONSHINYAKUCOL(OTCMKTS:NPNKF).
Pulmonaryarterialhypertensionisoneofabroadgroupofdisordersjointlydescribedaspulmonaryhypertension(PH).PAHcausesveryhighbloodpressureinthearteriesextendingbetweentheheartandlungs.Itisachronic,lethaldisorderwithsymptomssuchastirednessandlossofbreathandheartfailure.Itismoreprevalentinnon-Hispanicblacks,women,andpeopleaged75andover.
PAHisararediseaseaffectingonly15-50peoplepermillionaroundtheworld.However,theincidencerateishigherinhigh-riskpatientswouldbeindividualsinfectedwithHIV,whosuffera0.5%occurrencerate;patientswithsystemicsclerosishaveaprevalenceof7-12%,whilethediseaseaffectsaround2-3.75%patientswithsicklecelldisease..
Opsumit,worksasadualendothelinreceptorantagonist(ERA).It’ssafetyandefficacyofthedrugwasestablishedbySERAPHIN,thelargest,longestPAHfinal-stagetrialstudy.151centersinalmost40countrieswereusedtoundertakethestudy,whichwasconcluded2012.
Thetrialprimarilytestedmortalityandmorbidityratein287PAHpatientstreatedwitheithera3mgor10mgonce-dailydrug,Opsumitoraplacebopill.Theresultsindicatedthatthe10mgdoseofOpsumitreducedcombinedmortality-morbidityrateby45%ascomparedtotheplacebo.Thetrialresultsfurtherreportedatleast3%moreadverseeffectsinOpsumit-treatedpatientsthantheplacebogroup.Thecommonadverseeffectsincludedheadache,nasopharyngitis,anemia,urinarytractinfection,bronchitis,influenzaandpharyngitis.
TheSERAPHINprovidedthebasisoftheJapaneseapprovalforOpsumit.Thiswasfurtherbackedbypositiveresultsfromalocalstudyofthedrug.Inbothstudies,PAHaffectedvolunteerpatientsexhibitedpulmonaryvascularresistanceimprovements,a6minwalkdistance(6MWD),andWHOfunctionalclass.
TheoraldrugOpsumitbecomesthethirdmajordruginActelion’sPAHdrugportfolioalongsideEpoprostenol"ACT"andTracleer.TheintravenoustreatmentoptionforPAH,Epoprostenol“ACT”wasgrantedapprovalinJapanbackinFebruary2013.ThedrugismarketedinUSunderthebrandnameVeletri.TheoralendothelinreceptorantagonistTracleerhasbeenapprovedinJapansince2005.
Opsumit,however,hasprominenceovertheothertwoPAHdrugsunderActelion’sumbrella.Tracleerhasbeenknowntocauseabnormalenzymeelevationsintheliver,sothatpatientsarerequiredtoendurelivermonitoringeverymonth.Epoprostenol“ACT”hastobeinjectedwhichmakesitamorestablebutinconvenientPAHtreatmentoption.
SatoshiTanaka,PresidentofActelionJapan,describedOpsumit’simprovementoverActelion’spreviousPAHtreatments,"OpsumitrepresentsamajorstepforwardforthemanagementofPAHasthefirstandonlyapprovedPAHtreatmentwithprovenlong-termoutcomeefficacy.”
ActelionChiefExecutiveOfficer,Jean-PaulClozel,describedJapaneseapprovalofOpsumitas“agreatachievementforActelionJapan.”
inOctober2013OpsumitreceivedapprovalfromtheUSFoodandDrugAdministration,whilstEuropeanapprovalarrivedinDecember2013.andwasgiventhego-aheadinEuropeby.ThedrugiscurrentlybeinglaunchedintheAustralia,Canada,theEUSwitzerlandandtheUS.
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