通过创新促进健康：2025年新药疗法批准

Advancing Health Through Innovation:New Drug Therapy INNOVATION | PREDICTABILITY | ACCESS Contents Acting Director’s Message...........................................3Executive Summary...................................................5Innovation Across Medical Conditions...................................................5New Drugs for Patients with Rare Diseases............................................5 CDER’s Novel Drug Approvals of 2025..........................7 First-in-Class Drugs..................................................................................8Drugs for Rare Diseases...........................................................................9Other Novel Drug Approvals.................................................................11Fast Track................................................................................................13Breakthrough Therapy............................................................................14 Predictability: Meeting PDUFA Goals...........................16 Access: First Cycle Approvals First Cycle Approvals.............................................................................17Approval in the U.S. Before other Countries.........................................17 New Uses of Approved Drugs......................................18 Appendix A: CDER’s Novel Approvals of 2025...............30 Appendix B: Novel Drug Designations...........................34 Today, CDER releases its report, Advancing Health Through Innovation: New DrugTherapy Approvals 2025, the center’s 15th annual report summarizing notable The report highlights the year’s 46 novel drug approvals. They are considerednovel 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 in other countries, and exactlyhalf (23) received Orphan Drug Designation (intended to treat, diagnose or Tracy Beth Høeg,M.D., Ph.D. Acting Director,Center for DrugEvaluation and Research The number of novel drug and biologic approvals in 2025 was similar to theaverage for the last five years and above the historical average of 38 noveldrugs per year since 2007. Combined with CBER, there were 58 novel In total, CDER approved 123 products in 2025 (both novel drugs and non-novel drugs, including new dosage forms and formulations; this numberexcludes certain submission types, such as efficacy supplements). Thesedrugs will be used in a wide variety of medical areas, including infectious CDER also approved 18 biosimilars in 2025, 4 of which were for referenceproducts (existing FDA-approved biologics to which biosimilars are compared)that previously had no approved biosimilars. These include Merilog and Kirsty(a rapid-acting insulin for diabetes); Omlyclo (a treatment for asthma, chronicrhinosinusitis, food allergies, and chronic hives); and Poherdy (a treatment for Throughout the course of 2025, CDER continued to demonstrate its ongoingcommitment to ensuring safety and efficacy of all FDA approved products byissuing complete response (CR) letters for applications that did not meet FDA’sstatutory requirements for approval. In 2025, CDER issued 19 CR letters for18 novel drugs. As of September 2025, the FDA began making CR letters to It is worth noting that the work at CDER is not limited to the review of marketingapplications but also includes extensive engagement with companies throughoutdrug development, including providing advice on protocols and holding formalmeetings with sponsors. In 2025, CDER received over 3,000 INDs, comparable I would like to thank my CDER colleagues for their impressive work and theirpublic service over the past year. Their commitment to the scientific method,critical thinking and communication with companies is commendable. Above all, Tracy Beth Høeg, M.D., Ph.D.Acting Director, Center for Drug Evaluation and Research Executive Summary CDER approved many drug therapies in 2025. These approvals, spanning a widearray of diseases and conditions, aim to help people live better and potentially Innovation Across Medical Conditions In 2025, we approved 46 new drugs never before approved or marketed in theU.S., known as “novel” drugs. We also approved previously approved drugs for The 2025 actions, both novel and other important drug approvals, focus onprevention, diagnosis, treatment and cure in many different areas, includinginfectious disease; neurology; pain management; mental health; heart, blood,kidney and endocrine diseases; inflammation; lung diseases; allergies; New Drugs for Patients with Rare Diseases Patients with rare diseases are often in critical need of new therapies, as theseindividuals generally have few or no existing treatment options. In 2025, 23 of 46,or half, of our novel drug approvals received orphan drug designation because ●Barth syndrome, a serious mitochondrial disease that affects the hea