Here are brief reports on four oncology approvals by the U.S. Food and Drug Administration (FDA) in October 2025:
• Menin Inhibitor in NPM1-Positive AML : On October 24, 2025, the FDA approved revumenib (Revuforj), a menin inhibitor, for the treatment of relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation, in adult and pediatric patients 1 year and older who have no satisfactory alternative treatment options. Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700/AUGMENT-101). A susceptible NPM1 mutation was confirmed in all enrolled patients by using next-generation sequencing or polymerase chain reaction to look at the last exon of NPM1.
The rate of complete remission plus complete remission with partial hematologic recovery was 23.1% (95% confidence interval [CI] = 13.5%–35.2%), and the median duration was 4.5 months (95% CI = 1.2–8.1 months). Of the 46 patients dependent on red blood cell and/or platelet transfusions at baseline, 8 (17%) became independent of red blood cell and platelet transfusions during any 56-day postbaseline period.
The prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation and torsades de pointes, and embryofetal toxicity. The recommended revumenib dose varies by patient weight and concomitant use of strong CYP3A4 inhibitors.
• BCMA-Directed Antibody for Multiple Myeloma: On October 23, 2025, the FDA approved belantamab mafodotin-blmf (Blenrep), a B-cell maturation antigen (BCMA)–directed antibody and microtubule inhibitor conjugate, in combination with bortezomib and dexamethasone for the treatment of adults with relapsed or refractory multiple myeloma who have received at least two prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent.
Efficacy was evaluated in DREAMM-7, an open-label, multicenter trial in adults with relapsed or refractory multiple myeloma who had received at least one line of prior therapy. The trial excluded patients who were refractory or intolerant to daratumumab or bortezomib, had received prior BCMA-directed therapy, and had existing corneal disease (except for mild punctate keratopathy). Patients were randomly assigned 1:1 to receive either belantamab mafodotin, bortezomib, and dexamethasone (BVd) or daratumumab, bortezomib, and dexamethasone (DVd). The efficacy population included 217 patients (108 and 109 in respective arms) who had received at least two prior lines of therapy.
Efficacy was established based on progression-free survival and overall survival. The median progression-free survival was 31.3 months (95% CI = 23.5 months to not reached) in the BVd arm and 10.4 months (95% CI = 7–13.4 months) in the DVd arm (hazard ratio [HR] = 0.31; 95% CI = 0.21–0.47). The median overall survival was not reached and 35.7 months (95% CI = 21.1 months to not reached) in respective arms (HR = 0.49; 95% CI = 0.32–0.76).
Because of the risk of ocular toxicity, belantamab mafodotin is available only through a Risk Evaluation and Mitigation Strategy (REMS), called the BLENREP REMS. Other warnings and precautions include thrombocytopenia and embryofetal toxicity.
• PD-1 Inhibitor for Skin Cancer: On October 8, 2025, the FDA approved cemiplimab-rwlc (Libtayo) for the adjuvant treatment of adults with cutaneous squamous cell carcinoma at high risk of recurrence after surgery and radiation therapy. Efficacy was evaluated in C-POST, a randomized, double-blind, multicenter, placebo-controlled trial in 415 patients with cutaneous squamous cell carcinoma at high risk of recurrence after surgery and radiation therapy. Patients were required to complete adjuvant radiation therapy within 2 to 10 weeks of randomization. Patients were randomly assigned 1:1 to receive cemiplimab or placebo.
The major efficacy outcome measure was disease-free survival (defined as the time from randomization to the first documented disease recurrence by investigator assessment or death due to any cause). Median disease-free survival was not reached in the cemiplimab arm (95% CI = not evaluable to not evaluable) and 49.4 months (95% CI = 48.5 months to not evaluable) in the placebo arm (hazard ratio = 0.32, 95% CI = 0.20–0.51, P < .0001). The prescribing information includes warnings and precautions for immune-mediated adverse reactions, infusion-related reactions, complications of allogeneic hematopoietic stem cell transplantation, and embryofetal toxicity.
• Combination Therapy for Small Cell Lung Cancer: On October 2, 2025, the FDA approved lurbinectedin (Zepzelca) in combination with atezolizumab (Tecentriq) or atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) for maintenance treatment of adults with extensive-stage small cell lung cancer (SCLC) whose disease has not progressed after first-line induction therapy with atezolizumab or atezolizumab and hyaluronidase, carboplatin, and etoposide.
Efficacy was evaluated in IMforte, a randomized, multicenter, open-label trial in patients receiving first-line treatment for extensive-stage SCLC. In IMforte, 483 patients with extensive-stage SCLC whose disease had not progressed after completion of four cycles of atezolizumab, carboplatin, and etoposide (induction treatment) were randomly assigned 1:1 to receive either lurbinectedin in combination with atezolizumab administered intravenously (IV) or IV atezolizumab alone until disease progression or unacceptable toxicity.
The major efficacy outcome measures were overall survival and progression-free survival assessed by an independent review facility according to Response Evaluation Criteria in Solid Tumors version 1.1, measured from the time of randomization after completion of induction therapy. Median overall survival was 13.2 months (95% = 11.9–16.4 months) in the lurbinectedin with atezolizumab arm and 10.6 months (95% CI = 9.5–12.2 months) in the atezolizumab arm (HR = 0.73, 95% CI = 0.57–0.95; two-sided P = .0174). Median progression-free survival was 5.4 months (95% CI = 4.2–5.8 months) and 2.1 months (95% CI = 1.6–2.7 months) in the respective arms (HR = 0.54, 95% CI = 0.43–0.67; two-sided P < .0001).
The prescribing information for lurbinectedin includes warnings and precautions for myelosuppression, hepatotoxicity, extravasation resulting in tissue necrosis, rhabdomyolysis, and embryofetal toxicity. The prescribing information for atezolizumab and for atezolizumab and hyaluronidase includes warnings and precautions for severe and fatal immune-mediated adverse reactions, infusion-related reactions, complications of allogeneic hematopoietic stem cell transplantation, and embryofetal toxicity.
