The OncFive: Top Oncology Articles for the Week of 9/7

Welcome to OncLive®’s OncFive!

Every week, we bring you a quick roundup of the 5 top stories from the world of oncology—ranging from pivotal regulatory decisions to key pipeline updates to expert insights on breakthroughs that are moving the needle in cancer care. This resource is designed to keep you informed on the latest updates in the space, in just a matter of minutes.

Here’s what you may have missed this week:

FDA Approves TAR-200 in BCG-Unresponsive NMIBC With CIS

The FDA has approved the gemcitabine intravesical system (Inlexzo; formerly TAR-200) for adult patients with Bacillus Calmette Guérin (BCG)–unresponsive non–muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. The decision was supported by findings from cohort 2 of the phase 2b SunRISe-1 trial (NCT04640623), in which gemcitabine intravesical system as monotherapy (n = 83) elicited a confirmed complete response rate of 82% (95% CI, 72%-90%), with 51% of patients remaining in response for at least 1 year. The trial evaluated gemcitabine intravesical system as monotherapy and in combination with cetrelimab (JNJ-63723283) in patients with BCG-unresponsive high-risk NMIBC who refused or were ineligible for cystectomy, with development ultimately prioritized for monotherapy in the CIS population. In the safety population (n = 85), the most common adverse effects included urinary frequency, urinary tract infection, dysuria, and micturition urgency. Serious toxicities occurred in 24% of patients.

FDA Clears Selumetinib for Symptomatic Pediatric NF-1–Associated Inoperable Plexiform Neurofibromas

The regulatory agency also approved selumetinib (Koselugo) granules and capsules for pediatric patients aged 1 year and older with neurofibromatosis type 1 (NF-1) who have symptomatic, inoperable plexiform neurofibromas. The decision was supported by a bioavailability study in healthy adults and exposure matching between the phase 2 SPRINT trial (NCT01362803) in patients aged 2 years and older and the phase 1/2 SPRINKLE trial (NCT05309668) in patients aged 1 year and older. In SPRINT, selumetinib achieved an overall response rate (ORR) of 66% (95% CI, 51%-79%), with all patients experiencing partial responses and most maintaining them for at least 12 months. The recommended dose is 25 mg/m² orally twice daily until disease progression or unacceptable toxicity, with updated labeling reflecting broader pediatric safety data.

Relacorilant NDA Is Under FDA Review for Platinum-Resistant Ovarian Cancer

The FDA has accepted a new drug application seeking the approval of relacorilant in patients with platinum-resistant ovarian cancer and set a Prescription Drug User Fee Act decision date of July 11, 2026. The application was supported by findings from the phase 3 ROSELLA trial (NCT05257408) and earlier phase 2 studies. In ROSELLA, relacorilant plus nab-paclitaxel (Abraxane) led to a median progression-free survival (PFS) of 6.54 months vs 5.52 months with nab-paclitaxel alone (HR, 0.70; P = .0076). The combination also improved median overall survival (OS) to 15.97 months vs 11.50 months in the control arm (HR, 0.69; P = .0121). Safety findings showed that relacorilant was well tolerated, with AEs similar to those observed with nab-paclitaxel monotherapy.

Pirtobrutinib Monotherapy Meets Primary PFS End Point in Treatment-Naive CLL/SLL

Pirtobrutinib (Jaypirca) demonstrated a statistically significant improvement in PFS compared with bendamustine plus rituximab (Rituxan; BR) in treatment-naive chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) without 17p deletions, meeting the primary end point of the phase 3 BRUIN CLL-313 trial (NCT05023980). OS, a key secondary end point, favored pirtobrutinib, although the data were not yet mature; the primary OS analysis is planned for 2026. Safety results were consistent with prior trials of pirtobrutinib across treatment settings. Findings build on information from the phase 1/2 BRUIN trial (NCT03740529), which supported the 2023 FDA approval in relapsed/refractory CLL/SLL, and the phase 3 BRUIN CLL-321 (NCT04666038) and BRUIN CLL-314 (NCT05254743) studies. Detailed data from BRUIN CLL-313 will be shared at a medical congress and submitted for publication.

NCCN Adds Dordaviprone to Clinical Practice Guidelines in H3K27M-Mutant Diffuse High-Grade Glioma

The National Comprehensive Cancer Network has updated its Clinical Practice Guidelines in Oncology to include dordaviprone (Modeyso) as a category 2A single-agent option for pediatric and adult patients with recurrent or progressive diffuse high-grade glioma harboring an H3K27M mutation. The FDA granted accelerated approval to the agent in August 2025 for patients at least 1 year of age with diffuse glioma and an H3K27M mutation who experienced disease progression after prior therapy, with continued approval contingent on confirmation in the phase 3 ACTION trial (NCT05580562). The decision was supported by an integrated analysis of 50 patients from the phase 2 ONC006 (NCT02525692), phase 2 ONC013 (NCT03295396), phase 1 ONC014 (NCT03416530), phase 2 ONC018 (NCT03134131), and expanded-access ONC016 (NCT05392374) studies, which showed an ORR of 22% and a median duration of response of 10.3 months. Additional efficacy findings included a corticosteroid response rate of 46.7% and a median time to corticosteroid response of 3.7 months. In a pooled safety analysis of 376 patients, 33% experienced serious AEs, with common events including hydrocephalus, vomiting, headache, seizure, and muscular weakness.

Honorable Mention: The 22nd International Myeloma Society Annual Meeting, taking place September 17 to 20, 2025, in Toronto, Canada, will highlight key advances in multiple myeloma research. In exclusive interviews with OncLive, experts shared what they are most looking forward to seeing presented at the meeting.