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Here is your snapshot of all therapeutic options that were approved by the FDA in May 2025 spanning tumor types.
FDA Approval Roundup: May
Below is your guide to all the oncologic therapeutic options cleared by the FDA in May 2025. The roundup provides everything you need to know, right at your fingertips—all the topline data that supported the decisions and expert insights contextualizing what they mean for clinical practice.
Indication: The regulatory agency granted accelerated approval to the combination of avutometinib and defactinib (Avmapki Fakzynja Co-pack) for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.
Supporting Data: The decision was based on data from the phase 2 RAMP-201 trial (NCT04625270), in which the combination demonstrated a confirmed overall response rate (ORR) of 44% (95% CI, 31%-58%), including a 3.5% complete response rate and a 40% partial response (PR) rate. The duration of response (DOR) ranged from 3.3 to 31.1 months. The regimen was generally well tolerated, with serious adverse effects (AEs) reported in 32% of patients and treatment discontinuation due to toxicity occurring in 14% of patients.
Bradley Monk, MD, FACOG, FACS
Significance: This is the first FDA-approved treatment for LGSOC, addressing a major unmet need in a rare, recurrent, and historically treatment-resistant subtype of ovarian cancer. The regimen offers a targeted therapy option for patients with KRAS mutations and is expected to become a new standard of care, pending confirmation from the ongoing phase 3 RAMP 301 trial (NCT06072781).
“This is the first time we have ever had an FDA approval for LGSOC, a rare variant of ovarian cancer. Generally, we think of ovarian cancer as being high grade serous, but low-grade serous cancer is a [disease subtype] where 70% of the tumors are driven by RAS and [are] part of the MAPK pathway,” Bradley Monk, MD, FACOG, FACS, of Florida Cancer Specialists & Research Institute, told OncLive. “[Approximately] 30% of those tumors are KRAS mutant, [and] there are other mutations—including NRAS, BRAF, NF1, and other RAS pathway–associated gene mutations. However, KRAS mutations are particularly important, and this accelerated approval is [for agents targeting] tumors with KRAS mutations.”
We also spoke with Rachel N. Grisham, MD, of Memorial Sloan Kettering Cancer Center Westchester, about the significance of the approval:
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Ritu Salani, MD, MBA
Indication: TheHIF-2α inhibitor belzutifan (Welireg) was cleared by the FDA for the treatment of adult and pediatric patients aged 12 years or older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma.
Supporting Data: The decision was based on findings from the phase 2 LITESPARK-015 trial (NCT04924075).In cohort A1 (n = 72), belzutifan achieved an ORR of 26% (95% CI, 17%-38%) with a median DOR of 20.4 months. Over half of the responders (53%) maintained a response for at least 12 months. The median time to response (TTR) was 11 months. Notably,32% of patients reduced antihypertensive medication use by at least 50% for at least 6 months, suggesting a meaningful impact on symptom control.
Significance: Belzutifan offers a targeted, non-surgical treatment option for a rare and difficult-to-treat group of tumors with limited approved therapies. Its approval builds on the agent’s growing role across Von Hippel-Lindau (VHL)–associated and clear cell renal cell carcinoma (RCC) indications. The durable responses and potential for blood pressure stabilization represent clinically meaningful benefits for patients with advanced pheochromocytoma or paraganglioma.
“The fact that we now have an FDA-approved drug option that was prospectively evaluated in a very strong clinical trial is great, and I think it will mean a lot to patients and providers of this patient population,” Kimberly Perez, MD, of Dana-Farber Cancer Institute, told OncLive. She further discussed the clinical implications of the approval:
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Indication: The FDA granted accelerated approval to telisotuzumab vedotin-tllv (Emrelis) for adult patients with locally advanced or metastatic, nonsquamous non–small cell lung cancer (NSCLC) with high c-MET protein overexpression, defined as immunohistochemistry 3+ staining on at least 50% of tumor cells, who have received a prior systemic therapy.
Supporting Data: Approval was based on the phase 2 LUMINOSITY trial (NCT03539536), in which patients with high c-MET protein expression (n = 84) achieved an ORR of 35% (95% CI, 24%-46%), with a median DOR of 7.2 months (95% CI, 4.2-12). All responses were PRs, and 21% of patients maintained their response for at least 12 months. Treatment was well tolerated overall; key AEs included peripheral neuropathy, fatigue, and peripheral edema.
Jonathan Goldman, MD
Significance: Telisotuzumab vedotin is the first FDA-approved therapy specifically targeting high c-MET protein overexpression in NSCLC, addressing a distinct subset of patients with limited treatment options. This decision adds to the expanding class of antibody-drug conjugates (ADCs) in lung cancer and offers a personalized therapy for patients with poor prognosis. Ongoing phase 3 evaluation in TeliMET NSCLC-01 (NCT04928846) will further define its role vs standard chemotherapy.
“We have observed a paradigm shift in oncology in recent decades toward personalized, biomarker-driven therapeutics, allowing for better selection and optimized treatment outcomes,” Jonathan Goldman, MD, of UCLA, stated in a news release. “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.”
Stephen V. Liu, MD
Indication: The FDA has approved retifanlimab-dlwr (Zynyz) in combination with carboplatin and paclitaxel as a first-line treatment for adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). The agent was also cleared as monotherapy for patients with locally recurrent or metastatic SCAC with disease progression on or intolerance to platinum-based chemotherapy.
Supporting Data: The combination indication was based on results from the phase 3 POD1UM-303/InterAACT2 trial (NCT04472429), which demonstrated a median progression-free survival (PFS) of 9.3 months (95% CI, 7.5-11.3) with retifanlimab plus chemotherapy vs 7.4 months (95% CI, 7.1-7.7) for placebo plus chemotherapy (HR, 0.63; 95% CI, 0.47-0.84; P = .0006). Median overall survival (OS) was 29.2 months (95% CI, 24.2-not evaluable) vs 23.0 months (95% CI, 15.1-27.9), respectively (HR, 0.70; 95% CI, 0.49-1.01; P = .0273). The combination led to an ORR of 56% (95% CI, 48%-64%) and a median DOR of 14.0 months (95% CI, 8.6-22.2). Serious AEs occurred in 47% of patients, most commonly sepsis (3.2%), pulmonary embolism (3.2%), diarrhea (2.6%), and vomiting (2.6%).
For the monotherapy indication, the phase 2 POD1UM-202 trial (NCT03597295) showed an ORR of 14% (95% CI, 8%-23%) and a disease control rate of 49% in patients previously treated with platinum-based chemotherapy. Serious AEs occurred in 40% of patients. The safety profile was consistent with that of PD-1 inhibitors.
Significance: Retifanlimab is the first PD-1 inhibitor approved for patients with SCAC, offering new treatment options in both the frontline and previously treated settings.
“This FDA approval [indicates] an immunotherapy drug for the first time, and [this is] an area where there is a significant unmet need,” Marwan Fakih, MD, of City of Hope, told OncLive. He further discussed the clinical relevance of the decision:
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Sheela Rao, MBBS, MD, FRCP
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