FDA Approvals in Oncology: July-September 2025 

October 2, 2025 by

To help our readers keep track of the cancer therapies approved by the U.S. Food and Drug Administration (FDA), understand their impact for patients, and put them in context of the current therapeutic landscape, Cancer Research Catalyst provides a quarterly review of the latest approvals in oncology from the FDA. 

As the summer wound down, the FDA issued eight approvals covering lung cancer, breast cancer, and pediatric brain cancer, among other cancer types and a genetic disorder associated with the growth of fibromas and an increased risk of developing other tumors. In all, the approvals between July and September 2025 introduced five new treatments, including a first-in-class therapy, and made three alternative drug delivery approaches available for certain existing therapies, providing additional accessibility for patients, including children.

A First-in-class Treatment for Brain Cancer 

Diffuse midline glioma (DMG) is a form of brain cancer that affects both children and adults, and often possesses the H3 K27M mutation that is linked to aggressive disease and poor prognosis. Until now, there were no FDA-approved treatments specifically for patients with H3 K27M-mutated DMG that typically responds poorly to standard chemoradiation.

  • Dordaviprone (Modeyso) received accelerated approval for patients 1 year and older with H3 K27M-mutated DMG that has progressed after prior therapy.  

This was the first FDA approval for dordaviprone, a first-of-its-kind targeted therapy that employs dual mechanisms to target the unique biology of H3 K27M-mutated DMG, which is often associated with dependency on dopamine signaling and dysregulated mitochondrial metabolism. In addition to inhibiting the D2/3 dopamine receptor often overexpressed in H3 K27M DMG, dordaviprone also triggers the overactivation of the mitochondrial enzyme ClpP, which can cause cancer cell death through protein cleavage.

Dordaviprone is a first-of-its-kind targeted therapy for both children and adults with with H3 K27M-mutated diffuse midline glioma.

First Time Approvals for Lung Cancer, Breast Cancer, and Multiple Myeloma 

Lung cancer accounts for more deaths each year in the United States than any other cancer type. Recently, two new therapies were granted accelerated approval for patients with the most common form, non-small cell lung cancer (NSCLC). The treatments target human epidermal growth factor receptor 2 (HER2) and epidermal growth factor receptor (EGFR), receptors that are often overexpressed or mutated in NSCLC in ways that enhance their activity and support tumor growth.

  • The HER2-blocking zongertinib (Hernexeos) received accelerated approval for the treatment of adult patients with non-squamous NSCLC that is unresectable or metastatic, has been treated with prior systemic therapy, and has activating mutations in the HER2 tyrosine kinase domain (TKD). 

Another HER2-targeting therapy, the antibody-drug conjugate trastuzumab deruxtecan (Enhertu), is already approved to treat these patients, but it is associated with significant side effects and must be administered intravenously. What distinguishes this new approval is that zongertinib appears effective at targeting a broader range of HER2 mutations, demonstrated a “very favorable safety profile,” and is a pill that can be taken orally by patients.

The approval of zongertinib was based on results from the first-in-human Beamion LUNG-1 clinical trial first presented at the AACR Annual Meeting 2025 by John V. Heymach, MD, PhD, of The University of Texas MD Anderson Cancer Center, and a member of the AACR Lung Cancer Task Force.  

“This potentially practice-changing approval of zongertinib would provide access to a highly efficacious treatment option with a manageable safety profile and would be the first oral therapy and only tyrosine kinase inhibitor approved for patients with HER2-mutated NSCLC,” Heymach explained in an AACR press release before zongertinib was approved.

Read our blog post to learn more about the clinical trial results, which were published in the New England Journal of Medicine, that led to this FDA approval. 

  • The targeted therapy sunvozertinib (Zegfrovy) received accelerated approval for the treatment of adult patients with locally advanced or metastatic NSCLC that has insertion mutations in exon 20 of the EGFR gene and has progressed on or after chemotherapy.

Sunvozertinib inhibits multiple mutant forms of EGFR that can drive hyperactive signaling that stimulates cancer cell growth. Similar to zongertinib, sunvozertinib also improves on existing EGFR-targeting therapies for lung cancer, demonstrating activity against EGFR-mutated lung cancers with the T790M mutation associated with acquired resistance to earlier EGFR tyrosine kinase inhibitors. Sunvozertinib’s discovery and early clinical testing were first reported in the AACR journal Cancer Discovery.

With the approvals of the two new lung cancer therapies mentioned above, the FDA also approved the Oncomine Dx Express Test as a companion diagnostic to detect HER2 and EGFR mutations in patients’ tumors, in order to determine their eligibility for treatment with zongertinib and sunvozertinib, respectively. 

Certain patients with breast cancer also have a new treatment option. 

  • The selective estrogen receptor degrader (SERD) imlunestrant (Inluriyo) was approved for the treatment of adults with advanced or metastatic breast cancer that expresses the estrogen receptor (ER), does not express HER2, possesses mutations in the ESR1 gene, and has progressed following at least one line of endocrine therapy. 

A “pure” ER blocker, imlunestrant destabilizes and promotes the degradation of the receptor—including its mutated form—and follows elacestrant (Orserdu) as the second SERD to be approved for patients with advanced, ER-positive, HER2-negative, ESR1-mutated breast cancers. Simultaneously, the FDA also approved the Guardant360 CDx assay as a companion diagnostic device to detect ESR1 mutations in patients’ breast tumors in order to determine their eligibility for treatment with imlunestrant.

Imlunestrant is an oral medication approved for patients with advanced, ER-positive, HER2-negative, ESR1-mutated breast cancers.

The approval of imlunestrant was based on results from the phase III EMBER-3 clinical trial presented at the 2024 San Antonio Breast Cancer Symposium (SABCS) by Komal Jhaveri, MD, of Memorial Sloan Kettering Cancer Center. During a General Session at SABCS 2024, Jhaveri highlighted the effectiveness of imlunestrant both alone and in combination with the CDK4/6 inhibitor abemaciclib (Verzenio).

For more information on the study, read our press release from SABCS 2024, and check out our blog post to learn more about imlunestrant and other molecule degraders in breast cancer. 

A fourth treatment that received its first FDA nod over the summer provides an additional therapy option for patients with multiple myeloma, a type of blood cancer. 

  • The bispecific T-cell engager linvoseltamab-gcpt (Lynozyfic) received accelerated approval for the treatment of adult patients with relapsed or refractory multiple myeloma previously treated with at least four lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. 

Linvoseltamab targets the B-cell maturation antigen (BCMA) overexpressed by multiple myeloma cells, and also binds T cells via CD3 in order to promote immune-mediated destruction of the cancer cells. It is the third BCMA-targeting bispecific T-cell engager to be approved by the FDA for these patients. 

Alternative Drug Delivery Methods 

Three other approvals expanded the ways in which patients can receive existing drugs that have already been approved for delivery by other means for a variety of cancers.

  • The chemotherapy-based gemcitabine intravesical system (Inlexzo) was approved for adults with non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors, that does not respond to Bacillus Calmette-Guérin (BCG) therapy. 

While patients with bladder cancer have been able to receive gemcitabine chemotherapy administered via urinary catheter, prior to this approval the treatment was delivered as a single bolus dose, which can lessen the drug’s retention time due to urine washing out the bladder along with residual drug. This new approval delivers gemcitabine continuously over multiple weeks, thus ensuring long-term exposure of the bladder cancer cells to the cytotoxic chemotherapy. 

Neurofibromatosis type 1 (NF1) is a genetic disorder that can cause painful plexiform neurofibromas, and increase patients’ risk of developing certain tumors throughout their lifetime. The FDA previously approved selumetinib (Koselugo) for pediatric patients with NF1, and a newly approved delivery method may enable effective treatment in even younger patients.

  • This quarter, the targeted therapy selumetinib was approved in both granule and capsule form for children 1 year and older who have NF1 that causes plexiform neurofibromas that are symptomatic and inoperable. 

The capsule form of selumetinib was previously approved for pediatric patients with NF1 who are 2 years and older. Now, younger patients, who may have trouble swallowing pills, can benefit from the new granule formation, which can be mixed with certain foods for easier intake. 

Plexiform neurofibromas arise from tissue that surrounds nerve cells.

Immune checkpoint inhibitors (ICIs), which enhance the immune system’s ability to recognize and eliminate cancer cells, have changed how doctors treat a variety of advanced solid tumors. In September, the FDA approved an alternative administration route for the ICI pembrolizumab (Keytruda), which was previously delivered exclusively via intravenous (IV) infusion.

This new delivery method, in which pembrolizumab is combined with an enzyme to improve tissue permeability, can be used to treat all solid cancers that were covered under previous pembrolizumab approvals. The subcutaneous injection formulation of pembrolizumab follows similar approvals for two other PD-1/PD-L1 ICIs, nivolumab (Opdivo) and atezolizumab (Tecentriq).

In the case of pembrolizumab, this new approval covers a wide range of patients, including those with cancers of the bladder, breast, cervix, colon and rectum, endometrium, esophagus, head and neck, kidney, liver, lung, skin (melanoma), and stomach. Federal statistics estimated that these cancers will collectively affect more than 1.2 million individuals in the United States in 2025.