The combination of lunresertib (a PKMYT1 inhibitor) and camonsertib (an ATR inhibitor) produced durable responses in heavily pretreated patients with advanced endometrial cancer and separately in patients with platinum-resistant ovarian cancer, in the gynecologic expansion cohort of the phase 1, first-in-human MYTHIC trial (ClinicalTrials.gov NCT04855656), according to data presented at the American Association for Cancer Research (AACR) 2025 annual meeting. In the endometrial-cancer cohort (n=27), the reported overall response rate was 25.9% and the 24-week progression-free-survival rate was 43%; in the platinum-resistant ovarian-cancer cohort (n=24), the reported overall response rate was 37.5% and the 24-week progression-free-survival rate was 45%. The FDA reportedly granted Fast Track designation to this combination for both CCNE1-amplified/FBXW7- or PPP2R1A-mutated platinum-resistant ovarian cancer and for the analogous endometrial-cancer population; this is sourced only to company/trade-press reporting, not a direct FDA-published record, and is presented here as a reported claim requiring direct regulatory corroboration before being treated as confirmed regulatory fact — the FDA does not publish a searchable public list of individual Fast Track grants, the same structural sourcing limitation already disclosed elsewhere on this site for investigational, trial-status-only records. No FDA approval exists for this combination.

Luveltamab tazevibulin (luvelta), an investigational antibody-drug conjugate targeting folate receptor alpha (FRα), produced objective responses in patients with recurrent, platinum-resistant ovarian cancer in the dose-optimization portion of the phase 2/3 REFRaME-O1 trial (ClinicalTrials.gov NCT05870748), according to data presented at the Society of Gynecologic Oncology (SGO) 2025 annual meeting. At the selected dosing regimen (5.2 mg/kg every 3 weeks with prophylactic growth-factor support for the first two cycles, then 4.3 mg/kg every 3 weeks; n=25), the reported overall response rate was 32% and the disease control rate was 96%. A direct ClinicalTrials.gov registry query confirms this trial’s real, current status is TERMINATED — the randomized phase 3 comparison against investigator’s-choice chemotherapy, described as forthcoming in the original March 2025 presentation, did not proceed. Public reporting (company statements, trade press) attributes this to a March 2025 strategic portfolio decision by the drug’s developer, Sutro Biopharma, to deprioritize further internal investment in this specific ovarian-cancer program while seeking an external development partner — not a reported safety or efficacy failure of the dose-optimization data itself, which remains the last real result this trial produced. No FDA approval exists for this drug.

The FDA approved zolbetuximab-clzb (Vyloy), a claudin 18.2 (CLDN18.2)-directed cytolytic antibody, in combination with fluoropyrimidine- and platinum-containing chemotherapy, as a first-line treatment for adults with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are CLDN18.2-positive. The approval was based on two randomized, double-blind, phase 3 trials: SPOTLIGHT (ClinicalTrials.gov NCT03504397; primary publication in The Lancet, DOI 10.1016/S0140-6736(23)00620-7, PMID 37068504) and GLOW (ClinicalTrials.gov NCT03653507). Both trials met their primary endpoint of progression-free survival, as well as the key secondary endpoint of overall survival, when zolbetuximab was added to chemotherapy compared with chemotherapy alone. The FDA simultaneously approved a companion diagnostic test (the VENTANA CLDN18 RxDx Assay) to identify CLDN18.2-positive patients eligible for this treatment.

The FDA granted full (traditional) approval to tisotumab vedotin-tftv (Tivdak), an antibody-drug conjugate directed at tissue factor, for adults with recurrent or metastatic cervical cancer whose disease progressed on or after chemotherapy — converting the drug’s original September 2021 accelerated approval, which had been based on earlier, single-arm trial data, into a full approval. The conversion was supported by the randomized, open-label, phase 3 innovaTV 301 trial (ClinicalTrials.gov NCT04697628; results reported in Annals of Oncology, DOI 10.1016/j.annonc.2023.10.029), which enrolled 502 patients with recurrent or metastatic cervical cancer who had received one or two prior systemic regimens. Patients randomized to tisotumab vedotin had a median overall survival of 11.5 months versus 9.5 months with chemotherapy (hazard ratio 0.70; P=0.0038) and a median progression-free survival of 4.2 months versus 2.9 months (hazard ratio 0.67; P<0.0001) — both statistically significant improvements over chemotherapy.

The FDA granted accelerated approval to tovorafenib (Ojemda), a type II RAF kinase inhibitor, for patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (pLGG, the most common type of childhood brain tumor) harboring a BRAF fusion or rearrangement, or a BRAF V600 mutation — the first systemic (whole-body) therapy ever approved specifically for this population. The approval was based on the single-arm, open-label, phase 2 FIREFLY-1 trial (ClinicalTrials.gov NCT04775485; primary publication in Nature Medicine, DOI 10.1038/s41591-023-02668-y, PMID 37978284), which enrolled patients aged 6 months to 25 years with relapsed or refractory disease and an activating BRAF alteration. In the 76-patient efficacy population, tumors shrank or resolved (an objective response) in about 67% of patients, with a median duration of response of 16.6 months.

The FDA approved eflornithine (Iwilfin), an oral inhibitor of the enzyme ornithine decarboxylase, to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (a cancer that develops from immature nerve cells, most common in young children) who had already shown at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy — the first FDA approval of any therapy specifically intended to reduce relapse risk in this population. The approval rested on an externally controlled trial design: a single-arm study, Study 3b (ClinicalTrials.gov NCT02395666), compared against an external control group drawn from a separate National Cancer Institute/Children’s Oncology Group-sponsored trial (Study ANBL0032), and supported by additional confirmatory evidence (FDA approval summary published in the Journal of Clinical Oncology, DOI 10.1200/JCO.24.00546, PMID 38917371). The FDA granted this approval priority review, breakthrough therapy, and orphan drug designations.

The FDA granted accelerated approval to zanidatamab-hrii (Ziihera), a bispecific HER2-directed antibody, for adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer — a group of rare, aggressive cancers that includes cholangiocarcinoma (bile duct cancer) and gallbladder cancer. The approval was based on cohort 1 of the multicenter, single-arm, phase 2b HERIZON-BTC-01 trial (ClinicalTrials.gov NCT04466891; primary publication in The Lancet Oncology, DOI 10.1016/S1470-2045(23)00242-5, PMID 37276871), which enrolled 62 patients with HER2-positive biliary tract cancer who had already received gemcitabine-containing chemotherapy — the trial population was 53% gallbladder cancer, 27% intrahepatic cholangiocarcinoma, and 19% extrahepatic cholangiocarcinoma. Zanidatamab produced a confirmed objective response rate of 41.3% (52% per independent central review, the basis cited for the FDA’s approval decision) with a median duration of response of 14.9 months.

The FDA granted accelerated approval to selpercatinib (Retevmo), an oral RET kinase inhibitor, for pediatric patients aged 2 and older with RET-altered metastatic thyroid cancer or other RET-altered solid tumors requiring systemic therapy — the first FDA approval of any targeted therapy specifically for pediatric patients under 12 with a RET alteration. The approval was based on LIBRETTO-121 (ClinicalTrials.gov NCT03899792), an international, single-arm, multicohort phase 1/2 trial; in the primary efficacy population of 25 patients aged 2 to 20 with locally advanced or metastatic RET-activated solid tumors that had not responded to available therapies, the confirmed objective response rate (by blinded independent review) was 48%. Selpercatinib works by blocking an overactive RET protein that certain thyroid cancers and other solid tumors depend on for growth.

The FDA granted traditional (full) approval to pirtobrutinib (Jaypirca), the first and only non-covalent (reversible) BTK inhibitor, for adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) previously treated with a covalent BTK inhibitor — converting the drug’s original December 2023 accelerated approval into a full approval on the strength of a randomized, confirmatory trial. The conversion was supported by the randomized, open-label, phase 3 BRUIN-CLL-321 trial (ClinicalTrials.gov NCT04666038; primary publication in the Journal of Clinical Oncology, DOI 10.1200/JCO-25-00166, PMID 40479620), which randomized 238 previously treated CLL/SLL patients. Progression-free survival — the trial’s primary endpoint — was significantly longer with pirtobrutinib: median 11.2 months versus 8.7 months with investigator’s choice of idelalisib plus rituximab or bendamustine plus rituximab (hazard ratio 0.58, P=.0105), a real, statistically significant improvement.

The U.S. FDA approved an expanded indication for trastuzumab deruxtecan (Enhertu) dated January 27, 2025, covering HR-positive, HER2-low or HER2-ultralow unresectable or metastatic breast cancer after progression on endocrine therapy — the exact scope of the approval per FDA materials; this scope must not be broadened to other breast cancer populations. Clinical evidence: the DESTINY-Breast06 trial. Primary publication: PMID 39282896, https://pubmed.ncbi.nlm.nih.gov/39282896/, DOI https://doi.org/10.1056/NEJMoa2407086. Trial registry: https://clinicaltrials.gov/study/NCT04494425. Interstitial lung disease/pneumonitis is a known safety concern associated with this drug class and should be discussed using the primary evidence/FDA materials’ own safety context, not omitted. [Identifiers as supplied by the governing task directive, attributed to independent verification outside this environment — NOT independently re-verified live here; format-validated only.]