October 2025 proved to be a significant month for the oncology landscape, marked by a wave of key regulatory decisions from the US FDA. This period saw important full and accelerated drug approvals across various cancer types, expanding treatment options for patients with both solid tumors and hematologic malignancies.
From new combination maintenance therapies for extensive-stage small cell lung cancer (ES-SCLC) and an adjuvant treatment for high-risk cutaneous squamous cell carcinoma (CSCC), to a crucial approval in relapsed or refractory acute myeloid leukemia (AML) with a specific mutation, the month delivered on several anticipated action dates.
Furthermore, the FDA continued to expedite the development of promising agents by granting several breakthrough therapy and fast track designations, signaling a robust pipeline of innovative oncology therapeutics on the horizon. This roundup delves into the most notable FDA news and designations that will shape cancer care moving forward.
FDA Accepts sBLA for T-DXd Plus THP in HER2+ Breast Cancer
On October 1, the FDA accepted the supplemental biologics license application (sBLA) for trastuzumab deruxtecan (T-DXd; Enhertu) followed by paclitaxel (Taxol), trastuzumab (Herceptin), and pertuzumab (Perjeta; THP) for the neoadjuvant treatment of adult patients with HER2-positive stage 2 or 3 breast cancer. A Prescription Drug User Fee Act (PDUFA) target action date of May 18, 2026, has been set.
FDA Fast-Tracks ETX-636 in PIK3CA-Mutant, HR+ Breast Cancer
Also on October 1, the FDA granted fast track designation to ETX-636, a pan mutant-specific allosteric PIK3CA inhibitor, for the treatment of patients with PIK3CA-mutant, HR-positive, HER2-negative advanced breast cancer.
FDA Approves Lurbinectedin/Atezolizumab for ES-SCLC Maintenance
On October 2, the FDA approved the combination of lurbinectedin (Zepzelca) and atezolizumab (Tecentriq) or atezolizumab and hyaluronidase-tqjs (Tecentriq Hybrezas) as a first-line maintenance treatment in patients with ES-SCLC whose disease has not progressed following first-line induction therapy with atezolizumab, carboplatin, and etoposide.
AMXT 1501 and DFMO Combination Achieve FDA Orphan Drug Designation
Also on October 2, the FDA granted orphan drug designation to AMXT 1501 in combination with difluoromethylornithine (Iwilfin; DFMO) for the treatment of patients with neuroblastoma.
Orca-T Earns FDA Priority Review in Heme Malignancies
On October 6, the FDA accepted for priority review the biologics license application of Orca-T, an investigational allogeneic T-cell immunotherapy, for the treatment of hematologic malignancies including acute myeloid leukemia, acute lymphoblastic leukemia, and myelodysplastic syndromes. The FDA has set a PDUFA target action date of April 6, 2026.
FDA Issues CRL to NDA for Dasynoc in CML and ALL
On October 8, the FDA issued a complete response letter to the new drug application of Daysnoc, a lower-dose, bioequivalent formulation of dasatinib (Sprycel), based on good manufacturing practice issues observed at Xspray’s contract manufacturer.
FDA Approves Cemiplimab for Adjuvant Cutaneous Squamous Cell Carcinoma
Also on October 8, the FDA approved cemiplimab (Libtayo) as adjuvant treatment in adults with high-risk CSCC.
WTX-124 Earns FDA Fast Track for Refractory Melanoma
The FDA granted fast track designation to the investigational therapeutic agent WTX-124 for the potential treatment of patients with locally advanced or metastatic cutaneous melanoma that has progressed following standard-of-care immunotherapy, also on October 8.
FDA Fast Tracks ADCE-D01 for Soft Tissue Sarcoma Treatment
On October 9, the FDA granted fast track designation to the antibody-drug conjugate (ADC) ADCE-D01 for the treatment of soft tissue sarcoma.
Ficerafusp Alfa Earns FDA Breakthrough Therapy Designation for HPV-Negative R/M HNSCC
On October 13, the FDA granted breakthrough therapy designation to the bifunctional antibody ficerafusp alfa (BCA101) in combination with pembrolizumab (Keytruda) for the first-line treatment of HPV-negative, recurrent or metastatic head and neck squamous cell carcinoma.
FDA Breakthrough Designation Signals New Therapeutic Avenue in R/R MCL
On October 14, the FDA granted breakthrough therapy designation to the investigational BCL-2 inhibitor sonrotoclax (BGB-11417) for the treatment of adult patients with relapsed or refractory mantle cell lymphoma following therapy with a BTK inhibitor and an anti-CD20 agent.
FDA Grants Fast Track to NG-350A for pMMR Rectal Cancer
Also on October 14, NG-350A, an intravenously delivered oncolytic immunotherapy, was granted fast track designation by the FDA for the treatment of mismatch repair-proficient (pMMR) locally advanced rectal cancer.
FDA Grants Orphan Drug Status for MNV-201 in Low-Risk MDS
On October 15, the FDA granted orphan drug designation to the investigational mitochondrial cell therapy MNV-201 for the treatment of myelodysplastic syndrome.
Immunotherapy EO2463 Receives FDA Fast Track for Follicular Lymphoma
On October 16, the FDA granted fast track designation to the novel immunotherapy EO2463 for the treatment of follicular lymphoma, backed by positive interim data from the ongoing phase 2 SIDNEY trial (NCT04669171).
FDA Accepts NDA for New Nilotinib Formulation in Chronic Myeloid Leukemia
On October 21, the FDA accepted the new drug application for XS003, a formulation referencing the TKI nilotinib (Tasigna), for treatment of chronic myeloid leukemia. A PDUFA target action date has been set for June 18, 2026.
FDA Prioritizes sBLA Review for Enfortumab Vedotin/Pembro in MIBC
Also on October 21, the FDA granted priority review to the sBLA of enfortumab vedotin-ejfv (Padcev) in combination with pembrolizumab, radical cystectomy, and standard pelvic lymph node dissection as perioperative treatment for patients with muscle-invasive bladder cancer who are ineligible for or decline cisplatin-containing chemotherapy.
FDA Grants Fast Track Status to MT-125 in Glioblastoma
On October 22, the FDA gave fast track designation to MT-125, a first-in-class dual small-molecule inhibitor, for the treatment of glioblastoma.
Novel ADC Pamlectabart Tismanitin Earns FDA Fast Track for Myeloma Treatment
On October 23, the FDA granted fast track designation to the novel amanitin-based ADC pamlectabart tismanitin (HDP-101) for treatment of patients with relapsed or refractory multiple myeloma.
FDA Approves Revumenib in Mutant NPM1 AML
On October 24, the FDA approved the application for revumenib (Revuforj) in the treatment of relapsed or refractory NPM1-mutant AML, supported by positive data from the phase 2 portion of the AUGMENT-101 trial (NCT04065399).
FDA Grants Breakthrough Therapy Status to Zenocutuzumab for Patients with NRG1 Fusion
Also on October 24, the FDA granted breakthrough therapy designation to zenocutuzumab (Bizengri) for the treatment of patients with advanced unresectable or metastatic cholangiocarcinoma harboring an NRG1 gene fusion. This designation was supported by results from the ongoing phase 2 eNRGy trial (NCT02912949).
FDA Fast Tracks Anti-HER2 Biparatopic ADC for Platinum-Resistant Ovarian Cancer
On October 27, the FDA granted fast track designation to JSKN003, a biparatopic HER2-targeting ADC, for the treatment of platinum-resistant ovarian cancer, regardless of HER2 expression.
Daraxonrasib Earns FDA Orphan Drug Designation in Pancreatic Cancer
Also on October 27, the oral, multiselective inhibitor daraxonrasib (RMC-6236) was awarded orphan drug designation by the FDA for the treatment of pancreatic cancer.
FDA Awards Orphan Drug Status to ZEN-3694 for NUT Carcinoma
The FDA granted orphan drug designation to ZEN-3694, a novel oral therapy, for the treatment of NUT carcinoma, also on October 27.
