Tag: FDA approval

  • FDA Closes Out 2025 With 16 Oncology Approvals in Six Weeks

    FDA Closes Out 2025 With 16 Oncology Approvals in Six Weeks

    The US Food and Drug Administration (FDA) closed out 2025 with 16 hematology/oncology approvals in just six weeks, marking one of the most active periods of the year. Issued between early November and mid-December, the decisions spanned hematologic malignancies and solid tumors moving new evidence into clinical practice.

    The approvals covered a wide range of disease settings and mechanisms of action, from CAR T-cell therapy and bispecific antibodies to antibody–drug conjugates, targeted small molecules, and formulation advances. Several actions formalized trends that have been building across major medical meetings, including the continued expansion of immune-based therapies in lymphoid malignancies, growing reliance on molecular selection, and increasing attention to treatment delivery, access, and convenience.

    Hematologic cancers were featured prominently in the regulatory surge. New approvals and label expansions extended options in multiple myeloma, marginal zone lymphoma, follicular lymphoma, chronic lymphocytic leukemia, light chain (AL) amyloidosis, and acute myeloid leukemia, reinforcing the FDA’s ongoing shift toward targeted and immune-based approaches across both indolent and aggressive disease. In several cases, accelerated approvals were converted to traditional approval, signaling regulatory confidence in longer-term efficacy and safety data.

    Solid tumor decisions rounded out the period, with notable activity in breast, bladder, lung, gastric, and prostate cancers. These approvals frequently paired established agents with new settings or combinations, underscoring the agency’s emphasis on biomarker-driven treatment and perioperative strategies, particularly in earlier lines of disease.

    With these approvals in place, the focus now shifts to ensuring that these advances are accessible and used appropriately in everyday practice.

    References:

    U.S. Food and Drug Administration. Oncology (Cancer)/Hematologic Malignancies Approval Notifications. FDA website. Updated 2025. Accessed December 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications

  • FDA Approves Liso-Cel for R/R MZL

    On December 4, 2025, the US Food and Drug Administration (FDA) approved the CD19/CD3-directed CAR T-cell therapy lisocabtagene maraleucel (Breyanzi) for adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received 2 or more prior lines of systemic therapy.

    The approval was supported by the MZL cohort of the open-label, multicenter, single-arm phase 2 TRANSCEND FL trial (NCT04245839). A total of 77 patients underwent leukapheresis, and 66 treated patients comprised the primary efficacy analysis set. Patients received a single lisocabtagene maraleucel infusion 2 to 7 days after lymphodepleting chemotherapy.

    The primary endpoint was overall response rate (ORR), assessed per Lugano criteria by an independent review committee. In the intention-to-treat population, the ORR was 84.4% (95% CI, 74.4%–91.7%), including a 55.8% complete response rate (95% CI, 44.1%–67.2%). Median duration of response had not been reached at the time of analysis.

    The safety profile reflected the known adverse-event spectrum of lisocabtagene maraleucel. The prescribing information contains boxed warnings for cytokine release syndrome (CRS) and neurologic toxicities. Additional risks include hypogammaglobulinemia, serious infections, prolonged cytopenias, secondary malignancies, and immune-effector-cell–associated hemophagocytic lymphohistiocytosis–like syndrome.

    References:

    US Food and Drug Administration. FDA approves lisocabtagene maraleucel for relapsed or refractory marginal zone lymphoma. Published December 4, 2025. Accessed December 6, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lisocabtagene-maraleucel-relapsed-or-refractory-marginal-zone-lymphoma

  • FDA Approves Epcoritamab with Rituximab and Lenalidomide for R/R FL

    On November 18, 2025, the US Food and Drug Administration approved epcoritamab-bysp in combination with rituximab and lenalidomide for adult patients with relapsed or refractory follicular lymphoma (FL). On the same day, the FDA also converted the prior accelerated approval of epcoritamab-bysp monotherapy in patients with FL who have received at least two prior lines of systemic therapy to full approval.

    The combination approval was supported by data from EPCORE FL-1 (Study M20-638; NCT05409066), a phase 3, open-label, randomized trial enrolling 488 patients with relapsed or refractory FL. Patients were randomized 1:1 to receive epcoritamab-bysp with rituximab and lenalidomide (R²) or R² alone. The coprimary endpoints were progression-free survival (PFS) and overall response rate (ORR) assessed by independent review per Lugano 2014 criteria. ORR was 89% (95% CI, 84–93) in the epcoritamab arm compared to 74% (95% CI, 68–79) in the control arm. Median PFS was not reached in the epcoritamab arm (95% CI, 21.9 months–not reached) and was 11.2 months (95% CI, 10.5–not reached) in the R² arm, corresponding to a hazard ratio of 0.21 (95% CI, 0.13–0.33; P < .0001).

    Treatment-related adverse events included cytokine release syndrome (CRS) in 24% of patients (12% serious) and immune effector cell-associated neurotoxicity syndrome (ICANS) in 0.8%. Serious adverse reactions occurred in 51% of patients, including serious infections in 28%. Common adverse events (≥20%) included injection-site reactions, CRS, fatigue, upper respiratory tract infections, rash, diarrhea, pyrexia, cough, and headache. Grade 3–4 cytopenias included neutropenia (~30%), anemia (~10%), and thrombocytopenia (~8%). Epcoritamab-bysp is administered subcutaneously with a cycle 1 step-up dosing regimen followed by weekly and then every 4-week maintenance dosing.

    This approval offers a fixed-duration, outpatient, chemotherapy-free treatment option for relapsed or refractory FL, a disease marked by inevitable relapse and treatment resistance. The addition of epcoritamab-bysp to R² provides a novel immunotherapeutic strategy with clinically meaningful improvements in PFS and response rates.

    References:

    US Food and Drug Administration. FDA approves epcoritamab-bysp for follicular lymphoma indications. FDA. Published November 18, 2025. Accessed November 18, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-epcoritamab-bysp-follicular-lymphoma-indications

  • New Interchangeable Pertuzumab Biosimilar Approved for HER2-Positive Breast Cancer

    On November 13, 2025, the US Food and Drug Administration approved pertuzumab-dpzb (Poherdy) as an interchangeable biosimilar to pertuzumab (Perjeta) for the treatment of HER2-positive breast cancer, consistent with the indications of the reference product.

    The approval was based on analytical, nonclinical, and clinical data establishing biosimilarity and interchangeability with the reference product. Evidence included a pharmacokinetic study and a comparative clinical trial in patients with HER2-positive breast cancer, demonstrating no clinically meaningful differences in safety, purity, or potency.

    No new safety signals were observed. The safety profile of pertuzumab-dpzb is expected to reflect that of pertuzumab, with common adverse reactions including diarrhea, alopecia, neutropenia, nausea, fatigue, rash, and peripheral neuropathy. Serious risks include left ventricular dysfunction and infusion-related reactions.

    Pertuzumab-dpzb is the first interchangeable biosimilar to pertuzumab approved in the United States. The interchangeable designation permits pharmacy-level substitution, where state law allows, and may increase access while reducing treatment costs for patients with HER2-positive breast cancer.

    Reference
    US Food and Drug Administration. FDA approves new interchangeable biosimilar to Perjeta. Published November 13, 2025. Accessed November 18, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-new-interchangeable-biosimilar-perjeta

  • Ziftomenib Approved for NPM1-Mutant R/R AML

    On November 13, 2025, the US Food and Drug Administration approved the menin inhibitor ziftomenib (Komzifti) for adult patients with relapsed or refractory acute myeloid leukemia (AML) harboring a susceptible NPM1 mutation and for whom no satisfactory alternative treatments are available.

    The approval was based on the phase 1/2 KOMET-001 trial (NCT04067336), an open-label, multicenter study enrolling 112 adults with relapsed or refractory AML and confirmed NPM1 mutation. In the phase 2 cohort (n = 92), the primary endpoint was the combined complete remission (CR) and CR with partial hematologic recovery (CRh) rate. Ziftomenib achieved a CR + CRh rate of 21.4% (95% CI, 14.2–30.2), including CR in 17.0% and CRh in 4.5% of patients. Median duration of CR/CRh was 5.0 months (95% CI, 1.9–8.1). Among 66 patients who were transfusion dependent at baseline, 21.2% achieved transfusion independence for at least 56 days; 26.1% of 46 patients who were transfusion independent at baseline maintained that status.

    Adverse reactions included differentiation syndrome (25% overall; 15% grade ≥3), febrile neutropenia (26%), anemia (20%), and thrombocytopenia (20%). QTc prolongation was not clinically significant. Three percent of patients discontinued ziftomenib due to treatment-related adverse events. The prescribing information includes warnings for differentiation syndrome, QTc prolongation, and embryo-fetal toxicity.

    Ziftomenib is the first targeted therapy approved specifically for NPM1-mutant AML, a subset representing approximately 25%–30% of adult AML cases. Responses were observed across subgroups regardless of prior venetoclax exposure or hematopoietic stem cell transplant. “In these heavily pretreated, relapsed/refractory patients, similar response rates were seen … regardless of multiple lines of therapy,” said Dr. Eunice Wang, KOMET-001 investigator (OncLive, November 2023).

    References:

    FDA announcement: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ziftomenib-relapsed-or-refractory-acute-myeloid-leukemia-npm1-mutation

  • FDA Approves Sunvozertinib for EGFR Exon 20 Insertion-Positive Metastatic NSCLC

    On July 2, 2025, the U.S. Food and Drug Administration (FDA) granted accelerated approval to sunvozertinib for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring epidermal growth factor receptor (EGFR) exon 20 insertion mutations, (as detected by an FDA-approved test), whose disease has progressed on or after platinum-based chemotherapy.

    Sunvozertinib is an oral EGFR tyrosine kinase inhibitor. The approval was based on the phase WU-KONG6 multicenter study evaluating sunvozertinib monotherapy in patients with previously treated EGFR exon 20 insertion–positive NSCLC. The objective response rate was 46% with a median duration of response of 11.1 months.

    US Food and Drug website. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sunvozertinib-metastatic-non-small-cell-lung-cancer-egfr-exon-20. Accessed July 9, 2025.