Diving Into Every FDA Drug Approval for Cancer Care During May 2025

Recap of every FDA approval announced by the regulatory agency in the month of May 2025, spanning various cancer types.

During the month of May 2025, the U.S. Food and Drug Administration (FDA) announced their approval for several therapeutics and combination therapies in the field of oncology across several indications including anal, lung and ovarian cancers, as well as several others.

Here is a list of cancer therapies approved by the regulatory agency over the last month.

Zynyz and Chemotherapy for Some With Anal Cancer

On May 15th, the FDA approved first-line treatment with Zynyz (retifanlimab-dlwr) in combination with chemotherapy for the treatment of adult patients with inoperable, locally recurrent or metastatic squamous cell carcinoma of the anal canal. Notably, the FDA-approved chemotherapy regimen consists of platinum-based carboplatin and paclitaxel.

Additionally, Zynyz was also approved on the same day as a single-agent-treatment for the same patient population following disease progression on or intolerance to platinum-based chemotherapy.

"The FDA approval of Zynyz marks a pivotal moment, bringing effective combination and monotherapy treatment options to patients with advanced anal cancer after decades of limited innovation," CEO of Incyte, Hervé Hoppenot, said in a news release announcing the FDA approval. "At Incyte, we focus our efforts where we can make the biggest impact for patients. I am proud of our scientists and development teams for their perseverance in delivering the first approved PD-1 inhibitor to U.S. patients with [squamous cell carcinoma of the anal canal].”

The FDA approval of Zynyz was based on data from two trials: the POD1UM-303/InterAACT2 phase 3 study and the POD1UM-202 trial phase 2 study, which investigated the agent in combination with chemotherapy and as a monotherapy.

A clinically meaningful 37% reduction in the risk of disease progression or death with the combination of Zynyz and chemotherapy was seen in the POD1UM-303/InterAACT2 trial. Median progression-free survival was 9.3 months among those receiving the combination compared with 7.4 months in the placebo group. An interim analysis also revealed a 6.2-month improvement in median overall survival, with follow-up still ongoing.

Serious side effects occurred in 47% of patients treated with Zynyz and chemotherapy, and the most commonly reported were sepsis (3.2%), pulmonary embolism (3.2%), diarrhea (2.6%) and vomiting (2.6%).

Emrelis in Previously Treated, Locally Advanced or Metastatic, Non-Squamous NSCLC

On May 14th, the FDA approved treatment with Emrelis (telisotuzumab vedotin-tllv) in adult patients with previously treated, locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC), who also have high c-Met protein overexpression. According to the FDA approval, in this case, high c-Met protein overexpression is defined as 50% or more of tumor cells with strong (3+) staining, as determined by an FDA-approved test.

“We have observed a paradigm shift in oncology in recent decades toward personalized, biomarker-driven therapeutics, allowing for better selection and optimized treatment outcomes. People with c-Met overexpressing NSCLC have poor prognosis and limited treatment options, and Emrelis is a first-in-class ADC that can address a critical unmet need for this patient population," Dr. Jonathan Goldman, the associate director of Early Drug Development at UCLA Health, stated in a news release about the regulatory approval.

Goldman is also a professor of Medicine in the Hematology/Oncology Division and the director of Clinical Trials in Thoracic Oncology at UCLA, UCLA Health, as well as the chair of the University of California Lung Cancer Consortium.

This regulatory approval marks Emrelis as the first and only antibody drug conjugate approved by the FDA for patients with previously treated, advanced NSCLC who have high c-Met protein overexpression, which is important as this aggressive disease often leads to a poor prognosis and limited treatment options.

Welireg in Pediatric Patients With Locally Advanced, Unresectable or Metastatic PPGL

On May 14th, the regulatory agency approved treatment with Welireg (belzutifan) among those with locally advanced, unresectable or metastatic pheochromocytoma or paraganglioma (PPGL), specifically, in pediatric patients 12 years and older.

Notably, this regulatory approval — which was based off trial findings from the LITESPARK-015 study — marks the first FDA approval of an oral therapy for the treatment of patients with PPGL.

LITESPARK-015 evaluated treatment with Welireg for 72 patients in cohort A1, in which, the overall response rate was 26%, and responses lasted a median of 20.4 months. Of note, 60 patients were taking blood pressure medication in this treatment cohort, and in this group, 32% were able to reduce at least one of their medications by half for six months or longer.

Up to 25% of patients experienced side effects with treatment, which consisted of anemia, fatigue, muscle and joint pain, low lymphocyte and white blood cell counts. Moreover, elevated liver enzymes, calcium, potassium and alkaline phosphatase was observed in lab values. Shortness of breath, headache, dizziness and nausea were also reported.

Avmapki Fakzynja Co-Pack in KRAS+, Recurrent, Low-Grade Serous Ovarian Cancer

On May 8th, the FDA approved treatment with the Avmapki Fakzynja Co-pack (a combination of avutometinib and defactinib) in patients who have received prior systemic therapy for their KRAS-mutated, recurrent, low-grade serous ovarian cancer.

The open-label, multicenter RAMP-201 trial evaluated the efficacy of the investigative treatment in 57 adults, and ultimately, this investigation led to the FDA Approval. Among the patients observed, in a blinded review using standard criteria, the overall response rate was 44% and the duration of response was 3.3 to 31.1 months.

All participants had measurable disease and had received at least one prior systemic therapy, including platinum-based treatment. Notably, KRAS mutations were confirmed through local tumor testing. In accordance with the approved regimen, patients orally took 3.2 milligrams of avutometinib twice weekly and 200 milligrams of defactinib twice daily during the first three weeks of each four-week treatment cycle.

Treatment continued until disease progression or unacceptable toxicity. This dosing regimen evaluated in the clinical trial is considered the recommended dosing schedule.

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