FDA 2025 Cancer Drug Approvals: targeted therapy dominates

Abstract

This commentary discusses the FDA’s drug approvals in 2025, with a particular focus on cancer therapies and the role of companion diagnostics (CDx). Cancer has emerged as the leading therapeutic area, accounting for 35% of all new drug approvals, largely driven by targeted therapies, with kinase inhibitors representing nearly half of these drugs. Many of the drugs have received orphan drug designations and/or have utilized the Accelerated Approval Program. A key finding was the widespread adoption of the drug-diagnostic co-development model, in which a CDx assay is developed along with the drug and used for patient selection in clinical trials. However, a significant challenge is the frequent lack of concurrent drug and CDx assay approvals. The absence of an analytically and clinically validated CDx assay may pose a challenge for healthcare providers in accurately identifying eligible patients, potentially delaying access to appropriate therapy. The FDA’s cancer drug approvals for 2025 highlight an ongoing commitment to precision medicine, with several new targeted treatments, such as antibody-drug conjugates and kinase inhibitors, where CDx assays play an important role in identifying the appropriate patient population.

Keywords

FDA / drug approvals / oncology / hematology / precision medicine / biomarkers / companion diagnostics / drug development

References

[1] Developing and Labeling In vitro Companion Diagnostic Devices for a Specific Group of Oncology Therapeutic Products [Internet]. FDA; [cited 2026 Jan 23]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/developing-and-labeling-in-vitro-companion-diagnostic-devices-specific-group-oncology-therapeutic

[2] Slamon DJ, Leyland-Jones B, Shak S, Fuchs H, Paton V, Bajamonde A, et al. Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. N Engl J Med. 2001; 344: 783-92.

[3] Jørgensen JT, Winther H, Askaa J, Andresen L, Olsen D, Mollerup J. A Companion Diagnostic With Significant Clinical Impact in Treatment of Breast and Gastric Cancer. Front Oncol. 2021; 11: 676939.

[4] Ross JS, Slodkowska EA, Symmans WF, Pusztai L, Ravdin PM, Hortobagyi GN. The HER-2 receptor and breast cancer: ten years of targeted anti-HER-2 therapy and personalized medicine. Oncologist. 2009; 14: 320-68.

[5] List of Cleared or Approved Companion Diagnostic Devices (In Vitro and Imaging Tools) [Internet]. FDA; [cited 2026 Jan 23]. Available from: https://www.fda.gov/medical-devices/in-vitro-diagnostics/list-cleared-or-approved-companion-diagnostic-devices-in-vitro-and-imaging-tools

[6] Mullard A. 2025 FDA approvals. Nat Rev Drug Discov. 2026; 25: 81-7.

[7] Drugs@FDA: FDA-approved drugs [Internet]. FDA; [cited 2026 Jan 23]. Available from: https://www.accessdata.fda.gov/scripts/cder/daf/

[8] Search Orphan Drug Designations and Approvals [Internet]. FDA; [cited 2026 Jan 23]. Available from: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/

[9] Full Prescribing information for Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) injection, Revised: 11/2025 [Internet]. FDA; [cited 2026 Feb 1]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/761467s000lblOrig1.pdf

[10] Accelerated Approval Program [Internet]. FDA; [cited 2026 Feb 1]. Available from: https://www.fda.gov/drugs/nda-and-bla-approvals/accelerated-approval-program

[11] Fridlyand J, Simon RM, Walrath JC, Roach N, Buller R, Schenkein DP, et al. Considerations for the successful co-development of targeted cancer therapies and companion diagnostics. Nat Rev Drug Discov. 2013; 12: 743-55.

[12] Full Prescribing Information for Hyrnuo (sevabertinib), Revised: 11/2025 [Internet]. FDA; [cited 2026 Feb 14]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219972s000lblCorrected.pdf

[13] Jørgensen JT. Missing Companion Diagnostic for US Food and Drug Administration-Approved Hematological and Oncological Drugs. JCO Precis Oncol. 2022; 6: e2200100.

[14] Guidance for Industry and Food and Drug Administration Staff [Internet]. FDA; [cited 2026 Apr 8]. Available from: https://www.fda.gov/media/81309/download