2018年美国批准FDA上新药

1、NovelDrugApprovalsfor2018Innovationdrivesprogress.Whenitcomestoinnovationinthedevelopmentofnewdrugsandtherapeuticbiologicalproducts,FDA'sCenterforDrugEvaluationandResearch(CDER)supportsthepharmaceuticalindustryateverystepoftheprocess.Withitsunderstandingofthescienceusedtocreatenewproducts,testin

2、gandmanufacturingprocedures,andthediseasesandconditionsthatnewproductsaredesignedtotreat,FDAprovidesscientificandregulatoryadviceneededtobringnewtherapiestomarket.TheavailabilityofnewdrugsandbiologicalproductsoftenmeansnewtreatmentoptionsforpatientsandadvancesinhealthcarefortheAmericanpublic.Forthis

3、reason,CDERsupportsinnovationandplaysakeyroleinhelpingtoadvancenewdrugdevelopment.Eachyear,CDERapprovesawiderangeofnewdrugsandbiologicalproducts.Someoftheseproductsareinnovativenewproductsthatneverbeforehavebeenusedinclinicalpractice.Othersarethesameas,orrelatedto,previouslyapprovedproducts,andtheyw

4、illcompetewiththoseproductsinthemarketplace.Certaindrugsareclassifiedasnewmolecularentities("NMEs)forpurposesofFDAreview.ManyoftheseproductscontainactivemoietiesthathavenotbeenapprovedbyFDApreviously,eitherasasingleingredientdrugoraspartofacombinationproduct;theseproductsfrequentlyprovideimport

5、antnewtherapiesforpatients.SomedrugsarecharacterizedasNMEsforadministrativepurposes,butnonethelesscontainactivemoietiesthatarecloselyrelatedtoactivemoietiesinproductsthathavepreviouslybeenapprovedbyFDA.Forexample,CDERclassifiesbiologicalproductssubmittedinanapplicationundersection351(a)ofthePublicHe

6、althServiceActasNMEsforpurposesofFDAreview,regardlessofwhethertheAgencypreviouslyhasapprovedarelatedactivemoietyinadifferentproduct.FDA'sclassificationofadrugasan“NMEforreviewpurposesisdistinctfromFDA'sdeterminationofwhetheradrugproductisa“newchemicaentity"or"NCEzwithinthemeaningof

7、theFederalFood,Drug,andCosmeticAct.序号药品名活性物质批准日期FDA批准用途59Ultomirisravulizumab12/21/2018Totreatparoxysmalnocturnalhemoglobinuria(PNH)58Elzonristagraxofusp-erzs12/21/2018Totreatblasticplasmacytoiddendriticcellneoplasm(BPDCN)57Asparlascalaspargasepegol-mknl12/20/2018Totreatacutelymphoblasticleukemia(AL

8、L)inpediatricandyoungadultpatientsage1monthto21years56Motegrityprucalopride12/14/2018Totreatchronicidiopathicconstipation55Xospatagilteritinib11/28/2018Totreatpatientswhohaverelapsedorrefractoryacutemyeloidleukemia(AML)54Firdapseamifampridine11/28/2018TotreatLambert-Eatonmyasthenicsyndrome(LEMS)inad

9、ults53Vitrakvilarotrectinib11/26/2018Totreatpatientswhosecancershaveaspecificgeneticfeature(biomarker)52Daurismoglasdegib11/21/2018Totreatnewly-diagnosedacutemyeloidleukemia(AML)inadultpatients51Gamifantemapalumab-lzsg11/20/2018Totreatprimaryhemophagocyticlymphohistiocytosis(HLH)50Aemcolorifamycin11

10、/16/2018Totreattravelers'diarrhea49Yupelrirevefenacin11/8/2018Totreatpatientswithchronicobstructivepulmonarydisease(COPD)48Lorbrenalorlatinib11/2/2018Totreatpatientswithanaplasticlymphomakinase(ALK)-positivemetastaticnon-smallcelllungcancer47Xofluzabaloxavirmarboxil10/24/2018Forthetreatmentofacu

11、teuncomplicatedinfluenzainpatientswhohavebeensymptomaticfornomorethan48hours.46Talzennatalazoparib10/16/2018ForthetreatmentoflocallyadvancedormetastaticbreastcancerpatientswithagermlineBRCAmutation.45Tegsediinotersen10/5/2018Totreatpolyneuropathyofhereditarytransthyretin-mediatedamyloidosisinadults4

12、4Revcovielapegademase-lvlr10/5/2018TotreatAdenosineDeaminase-SevereCombinedImmunodeficiency(ADA-SCID)43Nuzyraomadacycline10/3/2018Totreatcommunity-acquiredbacterialpneumoniaandacutebacterialskinandskinstructureinfections42Seysarasarecycline10/1/2018Totreatinflammatorylesionsofnon-nodularmoderatetose

13、vereacnevulgarisinpatients9yearsofageandolder41Libtayocemiplimab-rwlc9/28/2018Totreatcutaneoussquamouscellcarcinoma(CSCC)40Vizimprodacomitinib9/27/2018Totreatmetastaticnon-small-celllungcancer39Eigalcanezumab-gnlm9/27/2018Forthepreventivetreatmentofmigraineinadults38Copiktraduvelisib9/24/2018Totreat

14、relapsedorrefractorychroniclymphocyticleukemia,smalllymphocyticlymphomaandfollicularlymphoma37Ajovyfremanezumab-vfrm9/14/2018Forthepreventivetreatmentofmigraineinadults36Lumoxitimoxetumomabpasudotox-tdfk9/13/2018Totreathairycellleukemia35Pifeltrodoravirine8/30/2018TotreatHIV-1infectioninadultpatient

15、s34Xeravaeravacycline8/27/2018Totreatcomplicatedintra-abdominalinfectionsinpatients18yearsofageandolder33Takhzyrolanadelumab8/23/2018TotreattypesIandIIhereditaryangioedema32Oxervatecenegermin-bkbj8/22/2018Totreatneurotrophickeratitis31Diacomitstiripentol8/20/2018TotreatseizuresassociatedwithDravetsy

16、ndromeinpatients2yearsofageandoldertakingclobazam30Galafoldmigalastat8/10/2018TotreattreatadultswithFabrydisease.29Annoverasegesteroneacetateandethinylestradiolvaginalsystem8/10/2018Newvaginalringusedtopreventpregnancyforanentireyear28Onpattropatisiran8/10/2018Totreatthepolyneuropathyofhereditarytra

17、nsthyretin-mediatedamyloidosisinadultpatients27Potelige0mogamulizumab-kpkc8/8/2018Totreattworaretypesofnon-Hodgkinlymphoma26Mulpletalusutrombopag7/31/2018Totreatthrombocytopeniainadultpatientswithchronicliverdiseasewhoarescheduledtoundergoaprocedure25Omegavenfishoiltriglycerides7/27/2018Asasourceofc

18、aloriesandfattyacidsinpediatricpatientswithparenteralnutrition-associatedcholestasis24Orilissaelagolixsodium7/23/2018Forthemanagementofmoderatetoseverepainassociatedwithendometriosis23Krintafe,tafenoquine7/20/2018Fortheradicalcure(preventionofrelapse)ofPlasmodiumvivaxmalaria22Tibsovoivosidenib7/20/2

19、018Totreatpatientswithrelapsedorrefractoryacutemyeloidleukemia21TPOXXtecovirimat7/13/2018Totreatsmallpox20Braftoviencorafenib6/27/2018Totreatunresectableormetastaticmelanoma19Mektovibinimetinib6/27/2018Totreatunresectableormetastaticmelanoma18Zemdriplazomicin6/25/2018Totreatadultswithcomplicatedurin

20、arytractinfections17Epidiolexcannabidiol6/25/2018Totreatrare,severeformsofepilepsy16Moxidectinmoxidectin6/13/2018TotreatonchocerciasisduetoOnchocercavolvulusinpatientsaged12yearsandolder15Olumiantbaricitinib5/31/2018Totreatmoderatelytoseverelyactiverheumatoidarthritis14Palynziqpegvaliase-pqpz5/24/20

21、18Totreatadultswitharareandseriousgeneticdiseaseknownasphenylketonuria(PKU)13Dopteletavatrombopag5/21/2018Totreatlowbloodplateletcount(thrombocytopenia)inadultswithchronicliverdiseasewhoarescheduledtoundergoamedicalordentalprocedure12Lokelmasodiumzirconiumcyclosilicate5/18/2018Totreathyperkalemia11A

22、imovigerenumab-aooe5/17/2018Forthepreventivetreatmentformigraine10Lucemyralofexidinehydrochloride5/16/2018Forthenon-opioidtreatmentformanagementofopioidwithdrawalsymptomsinadults9.Akynzeofosnetupitantandpalonosetron4/19/2018Topreventacuteanddelayednauseaandvomitingassociatedwithinitialandrepeatcours

23、esofhighlyemetogeniccancerchemotherapy8.Crysvitaburosumab-twza4/17/2018Totreatadultsandchildrenages1yearandolderwithx-linkedhypophosphatemia(XLH),arare,inheritedformofrickets7.Tavalissefostamatinib4/17/2018Totreatthrombocytopeniainadultpatientswithpersistentorchronicimmunethrombocytopenia(ITP)6.Ilum

24、yatildrakizumab3/20/2018Totreatadultswithmoderate-to-severeplaquepsoriasiswhoarecandidatesforsystemictherapyorphototherapy5.Trogarzoibalizumab-uiyk3/6/2018TotreatHIVpatientswhohavelimitedtreatmentoptions4.Erleadaapalutamide2/14/2018Totreatacertaintypeofprostatecancerusingnovelclinicaltrialendpoint3.Symdekotezacaftor;ivacaftor2/13/2018Totreatcysticfibrosisinpatientsage12yearsando