美国FDA药物评价和研究中心（CDER）：2025年度新药获批报告（英文版）（36页）.pdf

1、CENTER FOR DRUG EVALUATION AND RESEARCHAdvancing Health Through Innovation:New Drug Therapy Approvals 2025JANUARY 2026INNOVATION|PREDICTABILITY|ACCESSContentsActing Directors Message.3Executive Summary .5Innovation Across Medical Conditions .5New Drugs for Patients with Rare Diseases.5Efficiencies i

2、n Bringing Therapies to Market.6CDERs Novel Drug Approvals of 2025.7First-in-Class Drugs.8Drugs for Rare Diseases.9Other Novel Drug Approvals .11Fast Track.13Breakthrough Therapy.14Priority Review.14Accelerated Approval.15Overall Use of Expedited Development and Review Methods.15Predictability:Meeti

3、ng PDUFA Goals.16Access:First Cycle Approvals and First in U.S.Approvals.17First Cycle Approvals.17Approval in the U.S.Before other Countries.17New Uses of Approved Drugs.18Approved Drugs Expanded for New Pediatric Populations.22Biosimilar Approvals.25Other Important Approvals.27Conclusion.29Appendi

4、x A:CDERs Novel Approvals of 2025.30Appendix B:Novel Drug Designations.34Advancing Health Through Innovation:New Drug Therapy Approvals 2025 2Acting Directors MessageToday,CDER releases its report,Advancing Health Through Innovation:New Drug Therapy Approvals 2025,the centers 15th annual report summ

5、arizing notable drug approvals.The report highlights the years 46 novel drug approvals.They are considered novel because they have not previously been approved or marketed in the U.S.Of these,34 were new molecular entities(NMEs)and 12 were biologics.70%(32/46)were approved in the U.S.before approval

6、 in other countries,and exactly half(23)received Orphan Drug Designation(intended to treat,diagnose or prevent rare diseases that affect fewer than 200,000 people in the U.S.).Notable approvals included treatments for two rare mitochondrial diseases(Kygevvi for thymidine kinase 2 deficiency and Forz