Midyear Roundup: FDA’s Novel Oncology Drug Approvals in 2025

Each year, the FDA approves dozens of novel medicines that have never before been approved or marketed in the United States. These drugs are critical to patient populations impacted, who before these approvals often lacked a standard of care treatment option for their disease. Particularly in oncology, novel drugs are being developed at a rapid pace, expanding treatment capabilities for millions of patients with a form of cancer. It is critical for patients and providers alike to be aware of these novel drugs and their curative potential.¹

Below, Pharmacy Times^® provides a recap of the novel oncology drug approvals until June 2025, with related coverage from the Pharmacy Times website. The treatments are indicated for a range of diseases, including non-small cell lung cancer (NSCLC), acute myeloid leukemia, and ovarian cancer.

Image Credit: © Luciano Luppa - stock.adobe.com

Datopotamab deruxtecan-dlnk (Dato-DXd, Datroway; AstraZeneca, Daiichi Sankyo)

Approval Date: January 17, 2025

Indication: To treat adult patients with unresectable or metastatic, hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) (IHC 0, IHC1+, or IHC2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy.

Background: In the open-label, randomized phase 3 TROPION-Breast01 clinical trial (NCT05104866), Dato-DXd demonstrated favorable progression-free survival (PFS) and overall survival (OS) when compared with the investigator’s choice of standard single-agent chemotherapy. Dato-DXd also elicited improved objective response rate (ORR) and duration of response compared with chemotherapy alone.²

Related Coverage: FDA Approves Dato-DXd for Adults With Unresectable or Metastatic HR+, HER2- Breast Cancer

Treosulfan (Grafapex; Medexus Pharmaceuticals)

Approval Date: January 22, 2025

Indication: In combination with fludarabine, for use as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients aged 1 year and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Background: Treosulfan, an alkylating agent, indicated non-inferiority compared with busulfan (Myleran; Otsuka America Pharmaceutical, Inc) conditioning in patients with AML or MDS in the MC-FludT.14/L phase 3 clinical trial (NCT00822393). Two-year event-free survival was 64.0% in the treosulfan group and 50.4% in the busulfan group, demonstrating improved outcomes in patients treated with the regimen and highlighting its potential to become a standard option for allo-HSCT.³

Related Coverage: Treosulfan Improves Survival and Lowers Mortality Prior to Allo-HSCT in Patients With Myelofibrosis⁴

Vimseltinib (Romvimza; Deciphera Pharmaceuticals)

Approval Date: February 14, 2025

Indication: For the treatment of patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection could cause worsened functional limitations or severe morbidity.

Background: Vimseltinib is an investigational oral switch-control tyrosine kinase inhibitor designed to selectively inhibit colony-stimulating factor 1 receptor (CSF1R). TGCT, a rare disease caused by the translocation of the CSF1 gene, causes the development of benign tumors inside or near the joints of patients. Even after surgery, these tumors can return and cause tissue degeneration, necessitating novel non-surgical treatment options such as vimseltinib.⁵

Related Coverage: FDA Approves Vimseltinib for the Treatment of Adults With Symptomatic TGCT

Penpulimab-kcqx (Akeso Biopharma Co., Ltd.)

Approval Date: April 23, 2025

Indication: Used in combination with either cisplatin (Platinol; Bristol Myers Squibb) or carboplatin (Paraplatin; Bristol-Myers Squibb) and gemcitabine (Gemzar; Eli Lilly and Company) to treat adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC) or as a single agent while on or after platinum-based chemotherapy and at least 1 other prior line of therapy.

Background: Efficacy and safety of penpulimab-kcqx were evaluated in 2 phase 3 trials, AK105-304 (NCT04974398) and AK105-202 (NCT03866967). In each trial, whether in combination with chemotherapy or as a single agent, penpulimab-kcqx demonstrated positive results, including robust PFS (9.6 months) in AK105-304 and strong ORR (28%). Penpulimab is recommended to be dosed at 200 mg, either every 3 weeks when used with chemotherapy or every 2 weeks as a single agent, for a maximum of 24 months.⁶

Related Coverage: FDA Approves Penpulimab-Kcqx for Indications in Nonkeratinizing Nasopharyngeal Carcinoma

Avutometinib and Defactinib (Avmapki Fakzynja Co-Pack; Verastem Oncology)

Image Credit: © YURIMA - stock.adobe.com

Approval Date: May 8, 2025

Indication: To treat adult patients with KRAS-mutated recurrent, low-grade serous ovarian cancer (LGSOC) following receipt of prior systemic therapy.

Background: The combination of avutometinib, an MEK kinase inhibitor, and defactinib, an FAK inhibitor, provides a thorough blockage of signaling that drives the development of RAS/MAPK pathway-dependent tumors. With its approval, it became the first and only FDA-approved treatment for patients with LGSOC. The disease is a rare and persistent form of ovarian cancer that is highly recurrent and less sensitive to chemotherapy and hormone therapy, which is the current standard of care regimen for this population.⁷

Related Coverage: FDA Approves Avutometinib/Defactinib for Adults With KRAS-Mutated Recurrent LGSOC

Telisotuzumab vedotin-tllv (Emrelis; AbbVie)

Approval Date: May 14, 2025

Indication: For the treatment of locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression following the use of prior systemic therapy.

Background: This approval marks the first and only antibody-drug conjugate (ADC) that targets the c-Met protein in this patient population. ADCs are designed to deliver drugs such as telisotuzumab directly to the cancer cells expressing c-Met, which is a protein expressed at high levels in nearly 25% of advanced non-squamous NSCLC patients. Telisotuzumab was approved based on data from the ongoing phase 2 LUMINOSITY study (NCT03539536), which revealed a 35% ORR and median DOR of 7.2 months.⁸

Related Coverage: FDA Grants Accelerated Approval to Telisotuzumab vedotin-tllv for NSCLC

Taletrectinib (Ibtrozi; Nuavtion Bio Inc)

Approval Date: June 11, 2025

Indication: Approved for the treatment of adults with locally advanced or metastatic ROS1-positive (ROS1+) NSCLC.

Background: ROS1+ NSCLC is a rare, aggressive form of lung cancer. Taletrectinib is a central nervous system-active, selective, next-generation ROS1 inhibitor that was found to be effective against ROS1+ NSCLC in 2 multicenter, single-arm, open-label, phase 2 clinical trials called TRUST-I (NCT04395677) and TRUST-II (NCT04919811).⁹

Related Coverage: Taletrectinib Gains FDA Approval for Locally Advanced or Metastatic ROS1+ NSCLC

REFERENCES

1. FDA. Novel drug approvals for 2025. Last Updated June 17, 2025. Accessed June 18, 2025. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025

2. McGovern G. FDA Approves Dato-DXd for Adults With Unresectable or Metastatic HR+, HER2- Breast Cancer. Pharmacy Times. Published January 17, 2025. Accessed June 18, 2025. https://www.pharmacytimes.com/view/fda-approves-dato-dxd-for-adults-with-unresectable-or-metastatic-hr-her2--breast-cancer

3. Beelen DW, Trenschel R, Stelljes M, et al. Treosulfan or busulfan plus fludarabine as conditioning treatment before allogeneic haemopoietic stem cell transplantation for older patients with acute myeloid leukaemia or myelodysplastic syndrome (MC-FludT.14/L): a randomised, non-inferiority, phase 3 trial. Lancet Hematol. 2020;7(1):e28-e39. doi:10.1016/S2352-3026(19)30157-7

4. Gerlach A. Treosulfan Improves Survival and Lowers Mortality Prior to Allo-HSCT in Patients With Myelofibrosis. Pharmacy Times. Published October 7, 2024. Accessed June 18, 2025. https://www.pharmacytimes.com/view/treosulfan-improves-survival-lowers-mortality-prior-to-allo-hsct-in-patients-with-myelofibrosis

5. McGovern G. FDA Approves Vimseltinib for the Treatment of Adults With Symptomatic TGCT. Pharmacy Times. Published February 14, 2025. Accessed June 18, 2025. https://www.pharmacytimes.com/view/fda-approves-vimseltinib-for-the-treatment-of-adults-with-symptomatic-tgct

6. McGovern G. FDA Approves Penpulimab-Kcqx for Indications in Nonkeratinizing Nasopharyngeal Carcinoma. Pharmacy Times. Published April 24, 2025. Accessed June 18, 2025. https://www.pharmacytimes.com/view/fda-approves-penpulimab-kcqz-for-indications-in-nonkeratinizing-nasopharyngeal-carcinoma

7. McGovern G. FDA Approves Avutometinib/Defactinib for Adults With KRAS-Mutated Recurrent LGSOC. Pharmacy Times. Published May 8, 2025. Accessed June 18, 2025. https://www.pharmacytimes.com/view/fda-approves-avutometinib-defactinib-for-adults-with-kras-mutated-recurrent-lgsoc

8. Ferruggia K. FDA Grants Accelerated Approval to Telisotuzumab vedotin-tllv for NSCLC. Pharmacy Times. Published May 14, 2025. Accessed June 18, 2025. https://www.pharmacytimes.com/view/fda-grants-accelerated-approval-to-telisotuzumab-vedotin-tllv-for-nsclc

9. McGovern G. Taletrectinib Gains FDA Approval for Locally Advanced or Metastatic ROS1+ NSCLC. Pharmacy Times. Published June 11, 2025. Accessed June 18, 2025. https://www.pharmacytimes.com/view/taletrectinib-gains-fda-approval-for-locally-advanced-or-metastatic-ros1-nsclc