瑞士Santhera小分子药物idebenone获入FDA快速通道，杜氏肌营养不良患者有福了！

2015年4月1213日讯/生物谷BIOON/--瑞士Santhera制药公司治疗杜氏肌营养不良症（DMD）的药物Raxone/Catena（化学名:idebenone）三期临床试验结果甚是乐观，获得了FDA快速通道药物认定。
FDA快速通道药物提名旨在加速那些治疗严重疾病且很少获批上市的药物审批速度。专家分析，Santhera也可能获得该药物的优先审批权，这将进一步促使它加速上市。
艾地苯醌（idebenone）是一个化学合成的"短碳链苯醌"，刺激线粒体的电子传递，增加肌肉细胞中的能量供应。Idebenone同时也在美国和欧洲获得了孤儿症药物认证，在美国的专利保护期到2027年，欧洲到2026年。
对于广大杜氏肌营养不良患者来说，FDA授予Raxone/Catena快速通道地位无疑是一个巨大的好消息。杜氏肌营养不良是最常见的肌肉退行性病变，患者肌肉无力，可遗传。全球范围内，每3500名新生男婴儿中就有1名患有此症。杜氏肌营养不良极易引发早期死亡和高风险的心脏-呼吸衰竭，临床上唯一有效的治疗手段是糖皮质激素类固醇药物，类固醇有严重的副作用，不能长期服用。根据一项最新的报告，几乎42%或以上的超过10岁的DMD患者，都未接受过皮质类固醇治疗，或因类固醇的严重副作用而停止治疗。
此次FDA的决定是基于一项双盲、安慰剂对照III期研究DELOS的积极结果。该研究共招募了64名来自欧洲和美国未接受皮质类固醇激素治疗的10-18岁DMD患者，研究中，患者随机分配至idebenone（900mg/天）或安慰剂治疗52周，主要终点为从基线至治疗的52周，呼气流量峰值（PEF，又名最大呼气流量）占预测值百分比的变化（PEF%）。研究结果显示，在研究的第52周，安慰剂组PEF%相对基线显著下降（-9.01%p;95%CI:-13.2,-4.8;p＜0.001），idebenone治疗组PEF%相对基线下降不显著（-3.05%p;95%CI:-7.1,0.97;p=0.134）；2组PEF%差值为5.96%p（95%CI:0.16,11.8;p=0.044），表明idebenone使患者PEF%下降幅度显著改善66%。此外，在研究的26周（p=0.007）和39周（p=0.034）及横跨所有评估时间点（p=0.018），相对安慰剂组，idebenone治疗组均表现出统计学意义的显著疗效差异。
SantheraCEOThomasMeier十分看好idebenone，将全力打造该药物，使其成为公司长线收入的增长点。"我们将在未来几周内召开新药申请的文件准备会议，争取尽快向FDA提交。"
英文原文
SantheraPharmaceuticalsWinsFastTrackDesignationFromFDAForRaxone/CatenaTreatment
FDAsdecisiontoawardSantheraafasttrackdesignationforRaxone/Catenatreatmenthascomeonthebackofpositiveresultsfromalatestagetrial,DELOS
SwissspecialtypharmaceuticalcompanySantheraPharmaceuticals(OTCMKTS:SPHDF)hasfinallyachievedavictoryithadlongbeenstrivingfor.ThecompanysRaxone/CatenaregimenhaswonafasttrackdesignationfromtheUSFoodandDrugAdministration(FDA)fortreatingpatientssufferingfromDuchenneMuscularDystrophy(DMD).
Suchadesignationisboundtoexpeditetheregulatoryprocessforthedrug,openingdoorsforthefinalapprovaltocomesoon.FasttrackdesignationgrantedbyFDAismeantforaidingthedevelopmentandregulatoryreviewforthosedrugsaimedattreatingseriousdiseases;forwhichnootherorveryfewapprovedtreatmentsareavailableinthemarket.
ThedesignationalsoprovidesaidbyenablingpharmaceuticalcompaniestocommunicatewiththeFDAmorefrequently,whichsignificantlyhelpsinthedevelopmentandfinalapprovalprocessofthedrugs.Withthisfasttrackstatus,therearealsohighchancesthatSantheramightwinAcceleratedApprovalandPriorityReviewfromtheFDA,whichwillfurtherspeeduptheregulatoryprocess.
Raxone/Catena(chemicalnameidebenone),isa"syntheticshort-chainbenzoquinone,"whichworksbyenhancingmitochondrialelectrontransporttohaveasignificantimpactonincreasingtheenergylevelsofcellsfoundinmuscles.AddingtoSantherasdefinitesuccessinthefuture,IdebenonealsoenjoysorphandrugdesignationforDMD,bothintheUSandinEurope.IthasalsobeengivenpatentprotectionintheUStill2027,andinEuropetill2026.
TherecentFDAdecisiontograntfasttrackstatustoSantherasRaxone/CatenabringsgoodnewsforthelargenumberofpatientscurrentlylivingwithDMDacrosstheworld.DMD,consideredtobeagenetic,degenerativediseaseandthemostcommonandseriousformofmuscledegenerationthatleadstorapidweaknessofmuscles,iscurrentlyexperiencedbyoneinevery3,500malebabiesaroundtheworld.
Thedisease,whichresultsintohighearly-mortalityduetohighcardio-respiratoryfailureriskitentails,hastheonlyavailablemedicaltreatmentinformofglucocorticoidsteroids.Steroids,thoughreducethemusclesweakness,butalsocomewithserioussideeffects,whichsignificantlylimitstheiruseinDMDpatients.Almost42%ofpatientscurrentlylivingwithDMDaged10yearsormorehavenevertakensteroidsfortheirDMDtreatmentorhavestoppedtheiruseduetovarioussideeffects,asperdatagatheredinarecentstudy.
TheFDAsdecisionisduetopositiveresultsfromalatestagetrial,namedDELOS,whichwasadoubleblindandplacebo-controlledstudywithatotalof64enrollmentsofDMDpatientsfrombothEuropeandUS.TheresultsestablishedsignificantefficacyforSantherasregimen,whichprovedtofullymeettheprimaryendpointofthestudybyresultingintoasignificant66%ofreductioninlossofPeakExpiratoryFlowpercentfrombaselinetoweek52,whencomparedtothetreatmentgroupgivenaplacebo.Inaddition,Raxone/Catenaalsoledtoa"statisticallysignificanttreatmenteffect"whenobservedatweek26,39andacrossvariousdurationsthatwereassessed.
ThomasMeier,theCEOatSanthera,seemedverypositiveofthelong-termgrowthpotentialthattheRaxone/CatenaDMDtreatmentholdsforthecompany.Hestated:"WeareverypleasedthattheFDAhasgrantedFastTrackdesignationforRaxone/Catena,whichfurtherunderscorestheunmetmedicalneedforeffectivetreatmentsforpatientswithDMD."
Meanwhile,headded:"OnthebasisofthepositivedatafromourPhaseIIItrialwithRaxone/CatenainDMD,wehavestartedtoprepareaNewDrugApplicationandplantomeetwiththeFDAinthecomingweekstodiscussourNDAdossierinapre-NDAmeeting,"asperapressreleasebytheSwissdrugmakertoday.
Santherastockhasalreadygained15.34%,tradingatchf107.9inSixSwissStockExchange(SWX)asof12:10PMGMT+2.
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