Exploration of Targeted Anti-tumor Therapy

Table 1. Drugs for the treatment of oncological and hematological malignancies approved by the FDA in 2025 [6, 7].

Drug	OD/AA	Drug Class	Indication
Datopotamab deruxtecan	No/No	Antibody-drug conjugate	HR-positive, HER2-negative breast cancer
Treosulfan	Yes/No	Chemotherapeutic	Allogeneic hematopoietic stem cell transplantation in patients with AML/MDS
Mirdametinib	Yes/No	Kinase inhibitor	Neurofibromatosis type 1
Vimseltinib	No/No	Kinase inhibitor	Tenosynovial giant cell tumor
Penpulimab	Yes/No	Monoclonal antibody	Nonkeratinizing nasopharyngeal carcinoma
Avutometinib plus defactinib	Yes/Yes	Kinase inhibitors	KRAS-mutated low-grade serous ovarian cancer
Telisotuzumab vedotin	No/Yes	Antibody-drug conjugate	NSCLC with high c-Met protein overexpression
Taletrectinib	Yes/No	Kinase inhibitor	ROS1-Positive NSCLC
Linvoseltamab	Yes/Yes	Bispecific antibody	Multiple myeloma
Sunvozertinib	No/Yes	Kinase inhibitor	EGFR exon 20 insertion mutations in NSCLC
Dordaviprone	Yes/Yes	Small molecule inhibitor	H3 K27M mutated glioma
Zongertinib	No/Yes	Kinase inhibitor	HER2-mutated non-squamous NSCLC
Pembrolizumab & berahyaluronidase alfa	No/No	Monoclonal antibody plus an endoglycosidase	Multiple (see prescribing information for Keytruda Qlex [9])
Imlunestrant	No/No	Small molecule inhibitor	ER-positive, HER2-negative, ESR1-mutated breast cancer
Ziftomenib	Yes/No	Small molecule inhibitor	AML with NPM1 mutation
Sevabertinib	Yes/Yes	Kinase inhibitor	HER2-mutated non-squamous NSCLC

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OD, orphan drug; AA, accelerated approval; HR, hormone receptor; AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; NSCLC, non-small cell lung cancer.

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

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. [DOI]

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. [DOI] [PubMed] [PMC]

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. [DOI]

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

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

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

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

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. [DOI]

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. [DOI]

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

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