FDA 2025 年新药报告（中英文版）.pdfVIP

AdvancingHealthThroughInnovation:

NewDrugTherapyApprovals2025

通过创新推动健康：

FDA2025年新药疗法的批准

INNOVATIONPREDICTABILITYACCESS

创新可预测性可及性

FDA’sCenterforDrugEvaluationandResearch

FDA药品评审和研究中心

January2026

2026年1月

FDA2025年新药疗法的批准|iBIO(InternationalBiopharmaIndustryOrganization)2

CONTENTS|目录

ActingDirector’sMessage|代理主任寄语4

ExecutiveSummary|执行摘要8

InnovationAcrossMedicalConditions|创新覆盖多种疾病9

NewDrugsforPatientswithRareDiseases|罕见病新药9

EfficienciesinBringingTherapiestoMarket|上市流程效率提升10

CDER’sNovelDrugApprovalsof2025|2025年CDER创新药物批准情况12

First-in-ClassDrugs|首创新药15

DrugsforRareDiseases|罕见病药物17

OtherNovelDrugApprovals|其他新药批准情况20

Innovation:UseofExpeditedDevelopmentandReviewPathways|创新：加速开发与审评通道..22

FastTrack|快速通道23

BreakthroughTherapy|突破性疗法23

PriorityReview|优先审评24

AcceleratedApproval|加速批准25

OverallUseofExpeditedDevelopmentandReviewMethods|加速开发与审评方法的整体应用情

况26

Predictability:MeetingPDUFAGoals|可预测性：达成PDUFA目标27

Access:FirstCycleApprovalsandFirstinU.S.Approvals|可及性：首轮批准和在美首批29

FirstCycleApprovals|首轮审评通过30

ApprovalintheU.S.BeforeotherCountries|美国首先批准情况30

NewUsesofApprovedDrugs|已批准药物的新适应症31

ApprovedDrugsExpandedforNewPediatricPopulations|已批准药物扩展至新的儿童人群37

BiosimilarApprovals|生物类似药批准情况41

OtherImportantApprovals|其他重要批准45

Conclusion|结论49

AppendixA:CDER