接班重磅药Exjade诺华慢性铁过载口服新药Jadenu获FDA批准

2015年4月2日讯/生物谷BIOON/--瑞士制药巨头诺华（Novartis）近日宣布，FDA已批准新药Jadenu（deferasirox，地拉罗司）：（1）用于2岁及以上患者因输血治疗所致的慢性铁过载（CIO）的治疗；（2）用于10岁及以上非输血依赖型地中海贫血综合征（NTDT）患者慢性铁过载（CIO）的治疗。
Jadenu被开发作为诺华已上市药物Exjade（deferasirox）的接班人，该药在美国已上市10年，是处方量最大的一种铁螯合剂，年销售额近10亿美元。
Jadenu是Exjade的一种新口服配方，是首个可直接吞服的每日一次口服铁螯合剂。Exjade则是一种分散片，必须在液体中混合并在饭前至少30分钟空腹服用。Jadenu则在有无便餐的情况下均可直接吞服，从而大大简化了慢性铁过载（CIO）的治疗管理。Jadenu的上市，对于需要定期服用铁螯合剂治疗以清除体内过量铁的广大患者而言，是一个大好消息。
Jadenu的加速批准，是基于该药在临床试验中降低肝脏铁浓度和血清铁蛋白水平的疗效数据。目前，诺华正在开展临床研究进一步调查Jadenu的长期疗效和安全性。当前，许多镰状细胞病、地中海贫血或骨髓增生异常综合症患者需要频繁输血治疗，相应地也需要长期的日常铁螯合治疗。
慢性铁过载（CIO）是由于过量的铁长期在机体组织和器官（特别是心脏和肝脏）中积累所致，多见于频繁输血治疗的贫血患者，若不及时治疗可导致致命性的肝脏和心脏损伤。此外，慢性铁过载（CIO）也可能发展成一种名为血色素沉着症（hemochromatosis）的疾病，该病特征为虚弱、疲劳、关节或腹部疼痛甚至器官衰竭。因此，有效去除体内过量的铁，对于频繁接受输血治疗的患者而言极为重要。deferasirox（地拉罗司）是一种铁鳌合剂，用于改善铁过载疗效显著。
不过，需要注意的是，Jadenu也有相关的肾脏疾病风险，该药禁用于伴有严重肾脏或肝脏问题、晚期癌症、高危骨髓增生异常综合征(MDS)、过敏、低血小板计数的患者。除美国外，诺华也已向其他主要市场提交了Jadenu的监管申请。
英文原文：NovartisannouncesFDAapprovalforJadenuTMtosimplifytreatmentadministrationforpatientswithchronicironoverload
Jadenu(deferasirox),anewformulationofExjade(deferasirox),istheonlyonce-dailyoraltabletforironchelationJadenu,takenwithorwithoutfood,simplifiesdailytreatmentadministrationforpatientswithchronicironoverloadChronicironoverloadisaseriousconditionthatcanaffectpeoplewithsicklecelldisease,thalassemiaandmyelodysplasticsyndromesBasel,March30,2015-NovartisannouncedtodaythattheUSFoodandDrugAdministration(FDA)hasapprovedJadenuTM(deferasirox)tablets,aneworalformulationofExjade®(deferasirox)tabletsfororalsuspension,forthetreatmentofchronicironoverloadduetobloodtransfusionsinpatients2yearsofageandolder,andchronicironoverloadinnon-transfusion-dependentthalassemiasyndromes(NTDT)inpatients10yearsofageandolder.Jadenuistheonlyonce-dailyoralironchelatorthatcanbeswallowedwhole.
Manypatientswithsicklecelldisease,thalassemiaormyelodysplasticsyndromesneedrepeatedbloodtransfusionsandconsequently,long-termdailychelationtherapy.Jadenuoraltabletscanbetakeninasinglestep,withorwithoutalightmeal,simplifyingadministrationoftreatmentforchronicironoverload.Exjadeisadispersibletabletthatmustbemixedinliquidandtakenonanemptystomach.Jadenuisapprovedunderacceleratedapprovalbasedonareductionofliverironconcentrationsandserumferritinlevels.Continuedapprovalforthisindicationmaybecontingentuponverificationanddescriptionofclinicalbenefitinconfirmatorytrials.
"Novartishashadalong-termcommitmenttoimprovingthelivesofpatientswithchronicironoverload,"saidBrunoStrigini,President,NovartisOncology."Exjadetransformedironchelationtherapy.Werespondedtofeedbackfrompatientsandtheirphysicians,andnowJadenu,bysimplifyingtreatmentadministration,offersanimportantnewoptiontohelpmeetthesepatientsneeds."
Chronicironoverloadisalife-threateningcumulativetoxicitythatresultsfrombloodtransfusionsrequiredtotreatsicklecelldisease,myelodysplasticsyndromes,thalassemiaandotherconditions.ChronicironoverloadalsocanoccurinpatientswithNTDTduetoincreasedironabsorptioninthestomachandintestines[1],[2].Ifleftuntreated,chronicironoverloadcandamagetheliverandheart[3],[4].
Jadenucontainsdeferasirox,thesameactiveingredientthatisinExjade,amedicinethathasbeenusedbypatientswithchronicironoverloadforalmost10years.Exjadecurrentlyisthemost-prescribedchelatorintheUnitedStates[5].
"Jadenuisanexcitingdevelopmentforpatientswithchronicironoverloadwhohavebeeneagerforalternativetreatmentoptions,"saidDr.ElliottVichinsky,DirectorofHematologyandOncologyattheUniversityofCalifornia,SanFrancisco(UCSF)BenioffChildrensHospitalOaklandandProfessor,UCSFSchoolofMedicine."Takingironchelationtherapyeverydayhassometimesbeenachallengeforthem.TheadministrationofJadenuoraltabletsonceadayissimple."
NovartishassubmittedadditionalregulatoryapplicationsforJadenuinothercountriesworldwide.
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