FDA授予阿斯利康肿瘤免疫疗法tremelimumab孤儿药地位

2015年4月15日讯/生物谷BIOON/--英国制药巨头阿斯利康（AZN）肿瘤学管线近日收获了一个孤儿药地位，FDA已授予肿瘤免疫疗法tremelimumab治疗恶性间皮瘤（malignantmesothelioma，MM）的孤儿药地位。
间皮瘤是一种罕见的恶性肿瘤，预后极差，通常是由于接触石棉（asbestos）所致，最常影响肺和腹腔的衬里（lining），该病治疗选择非常有限，尤其是晚期疾病患者。目前，间皮瘤的5年生存率少于百分之五，甚至当疾病得到及时诊断和治疗时也是如此，大多数患者在确诊后一年内死亡。tremelimumab有望为该患者群体提供一种潜在的新治疗选择。
tremelimumab是一种抗细胞毒性T淋巴细胞抗原4（CTLA-4）全人源化单克隆抗体，能够阻断帮助肿瘤逃避免疫检查的信号通路。tremelimumab通过结合表达于活化的T淋巴细胞表面的CTLA-4蛋白，刺激机体免疫系统对肿瘤细胞发起攻击。
值得一提的是，百时美施贵宝的肿瘤免疫疗法Yervoy(ipilimumab,易普利姆玛)与tremelimumab同属于抗CTLA-4单抗，Yervoy已于2011年获FDA批准上市，用于晚期转移性黑色素瘤的治疗。
除了作为间皮瘤的一种单药疗法，目前阿斯利康正在多种类型肿瘤中探索tremelimumab与PD-L1免疫疗法MEDI4376组合的临床潜力，包括非小细胞肺癌（NSCLC）、头颈部癌症等。此外，阿斯利康也正在调查tremelimumab联合易瑞沙（Iressa）治疗EGFR突变型NSCLC，以及联合MEDI6469（一种小鼠OX40激动剂）治疗多种实体瘤。
英文原文：TremelimumabgrantedOrphanDrugDesignationbyUSFDAfortreatmentofmalignantmesothelioma
Wednesday,15April2015
AstraZenecatodayannouncedthattheUSFoodandDrugAdministrationhasgrantedOrphanDrugDesignationfortheanti-CTLA-4monoclonalantibody,tremelimumab,forthetreatmentofmalignantmesothelioma.
Mesotheliomaisarare,aggressivecancerthatmostoftenaffectstheliningofthelungsandabdomen.Availabletreatmentsformesotheliomaareverylimited,particularlyforpatientswithadvanceddisease.
“Thereisasignificantneedfornewtreatmentoptionsforpatientswithmesotheliomabecausefewerthanfivepercentofpatientscurrentlysurvivebeyondfiveyears,evenwhentheyreceivetimelydiagnosisandcare.Ouraimistorapidlyadvancethedevelopmentoftremelimumabasapotentialnewtreatmentoptionforthesepatients,”saidRobertIannone,SeniorVicePresident,HeadofImmuno-oncology,GlobalMedicinesDevelopmentatAstraZeneca.
TheOrphanDrugDesignationprogrammeprovidesorphanstatustodrugsandbiologics,whicharedefinedasthoseintendedforthesafeandeffectivetreatment,diagnosisorpreventionofrarediseasesordisordersthataffectfewerthan200,000peopleintheUS1.
Tremelimumabispartofthebroadpipelineofimmuno-oncologyassetsbeingdevelopedbyAstraZenecaanditsbiologicsresearchanddevelopmentarm,MedImmune,whicharedesignedtoharnessthebody’sownimmunesystemtofightcancer.Itisafullyhumanmonoclonalantibody,whichstimulatestheimmunesystemtodestroycancercellsthroughbindingtotheproteinCTLA-4,expressedonthesurfaceofactivatedT-lymphocytes.
Inadditiontobeinginvestigatedasamonotherapytreatmentforpatientswithmesothelioma,tremelimumabiscurrentlybeingstudiedincombinationwithAstraZeneca’santiPD-L1investigationalimmunotherapy,MEDI4736,intumourtypesincludingnon-smallcelllungcancerandheadandneckcancer.ItisalsobeingstudiedincombinationwithIressa(gefitinib)inEGFRmutatednon-smallcelllungcancerandwithMEDI6469(amurineOX40agonist)insolidtumours.
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