OncLive’s September Roundup of Key FDA Approvals in Oncology: 4 Decisions to Know

Below is your guide to all the oncologic therapeutic options that were greenlit by the FDA in September 2025. The roundup provides everything you need to know, right at your fingertips—all the topline findings that supported the regulatory decisions and expert insights contextualizing what they mean for clinical practice.

9/9: Gemcitabine Intravesical System in BCG-Unresponsive NMIBC With CIS

Indication: The FDA approved the gemcitabine intravesical system (Inlexzo) for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)–unresponsive non–muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors.

Supporting data: The regulatory decision was supported by data from cohort 2 of the phase 2b SunRISe-1 trial (NCT04640623), where Inlexzo monotherapy (n = 83) elicited a confirmed complete response (CR) rate of 82% (95% CI, 72%-90%). Notably, 51% of patients maintained a response for at least 1 year.

Clinical significance: This decision establishes the first sustained-release intravesical chemotherapy option for patients with BCG-unresponsive NMIBC. By offering a bladder-sparing alternative, Inlexzo provides a meaningful option for those who would otherwise face radical cystectomy in a setting with few advances over the past 40 years.

“This is a new drug that's available that works well and is going to be [an] effective therapy for patients going forward. This is a drug that's going to save a lot of bladders,” Joseph Jacob, MD, MCR, of State University of New York Upstate Medical University, said in an exclusive interview with OncLive®:

OTHER RELATED COVERAGE

• In a past interview, Jacob discussed the background and rationale for the SunRISe‑1 trial, the significance of the efficacy data, and considerations for incorporating the approach into clinical practice for patients with BCG‑unresponsive NMIBC.
• In another interview, Bogdana Schmidt, MD, MPH, of University of Utah Huntsman Cancer Institute, spotlighted novel treatment delivery systems under evaluation, including UGN-102 and TAR-200.
• In an exclusive recap of the top data to come out of the 2025 American Urological Association Annual Meeting, two presentations on TAR-200 from SunRISe-1 were spotlighted. Felix Guerrero-Ramos, MD, PhD, of Hospital Universitario 12 de Octubre, said: “Being focused on bladder cancer, because this is the pathology I treat every day, I am excited with all the data from cohort 4 of SunRISe-1, but we also have the data from cohort 2 of SunRISe-1, [which showed] an impressive CR rate for [patients with NMIBC with] CIS and a very significant duration of response, which is good for our patients.”
• At the meeting, Guerrero-Ramos further discussed the potential role of TAR-200 in high-risk, BCG-unresponsive, papillary-only NMIBC.
• In a prior interview, Sia Daneshmand, MD, of Keck Medicine of the University of Southern California, shared key efficacy and safety data with TAR-200 from SunRISE-1, how this therapy addresses an unmet need for this population, and directions for future research with TAR-200, including reinduction and therapeutic sequencing.

9/10: Selumetinib in Symptomatic Pediatric NF-1–Associated Inoperable Plexiform Neurofibromas

Indication: The next day, the regulatory agency cleared 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 (PNs). This expands the prior approval from 2020, which was limited to patients aged 2 years and older.

Supporting data: The decision was based on bridging data from a relative bioavailability study, the phase 2 SPRINT trial (NCT01362803) in patients aged 2 years or older, and the phase 1/2 SPRINKLE trial (NCT05309668) in patients aged 1 year or older. In SPRINT, selumetinib capsules (n = 50) induced an overall response rate of 66% (95% CI, 51%-79%), with all responses partial and 82% sustained for at least 12 months. Exposure-matching supported extrapolation of efficacy to younger children. The toxicity profile was consistent with prior data, with no new signals identified.

Clinical significance: This approval lowers the treatment age threshold to 1 year, providing earlier access to the first FDA-approved therapy for NF1-associated inoperable PNs. By addressing a high-burden, morbid condition in very young children, the expanded approval enables earlier intervention to manage disfigurement, pain, and functional impairment associated with NF1.

OTHER RELATED COVERAGE

• In September 2025, the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) recommended the approval of selumetinib for use in this population.
• In a previous OncLive On Air podcast episode, Christopher L. Moertel, MD, of the Katie Hageboeck Children’s Cancer Research Fund Clinic and the University of Minnesota School of Medicine, discussed the expansion of the NF1-associated PN treatment paradigm to include the MEK inhibitors selumetinib and mirdametinib (Gomekli); the benefits of offering treatment options in oral formulations; the toxicities associated with MEK inhibitors; and more.
• The primary end point of the phase 3 KOMET trial (NCT04924068) was met when selumetinib led to a statistically significant and clinically meaningful improvement in ORR vs placebo in adult patients with NF-1 who have symptomatic, inoperable PN.
• In June 2021, the European Commission granted a conditional approval to selumetinib for use in pediatric patients with symptomatic, inoperable PN and NF1 who are at least 3 years of age.

9/19: Subcutaneous Pembrolizumab in Solid Tumors

Indication: The FDA approved pembrolizumab and berahyaluronidase alfa-pmph (subcutaneous pembrolizumab; Keytruda Qlex) for subcutaneous injection in adult and pediatric patients aged 12 years or older across all solid tumor indications currently approved for intravenous (IV) pembrolizumab (Keytruda).

Supporting data: The decision was based on findings from the phase 3 3475A-D77 trial (NCT05722015), which demonstrated noninferior pharmacokinetics of subcutaneous vs IV pembrolizumab when combined with chemotherapy in patients with metastatic non–small cell lung cancer. Efficacy outcomes were comparable: ORR 45% (95% CI, 39%-52%) with subcutaneous vs 42% (95% CI, 33%-51%) with IV; median progression-free survival (PFS) was 8.1 vs 7.8 months, respectively; overall survival was not reached in either arm. Safety profiles were similar, with only mild injection-site reactions reported in the subcutaneous arm.

Clinical significance: The availability of a subcutaneous formulation offers a more convenient, less resource-intensive alternative to IV pembrolizumab while maintaining equivalent efficacy and safety. This approval broadens delivery options across all pembrolizumab solid tumor indications, potentially improving patient experience and infusion center efficiency.

“The approval of the subcutaneous form of pembrolizumab gives us another option that we can use to tailor treatments to [each] patient’s individual situation,” J. Thaddeus Beck, MD, FACP, of Highlands Oncology Group, told OncLive. Check out the rest of the clip:

OTHER RELATED COVERAGE

• In September 2025, the EMA’s CHMP issued 2 positive opinions for pembrolizumab, one being the subcutaneous formulation of pembrolizumab across all adult indications in the European Union.
• Data from the phase 3 3475A-D77 trial were shared during the 2025 European Lung Cancer Congress. Topline data had been shared in November 2024 and showed that subcutaneous pembrolizumab plus chemotherapy generated noninferior pharmacokinetics vs the IV formulation with chemotherapy in patients with previously untreated metastatic NSCLC.

9/25: Imlunestrant in ER+/HER2– ESR1-Mutated Metastatic Breast Cancer

Indication: The regulatory agency cleared imlunestrant (Inluriyo) for use in adult patients with estrogen receptor–positive, HER2-negative advanced or metastatic breast cancer harboring ESR1 mutations whose disease has progressed after 1 or more prior line of endocrine therapy.

Supporting data: The data were based on findings from the phase 3 EMBER-3 trial (NCT04975308). In patients with ESR1-mutated breast cancer (n = 256), imlunestrant reduced the risk of progression or death by 38% vs fulvestrant (Faslodex or exemestane (Aromasin; HR, 0.62; 95% CI, 0.46-0.82; P = .0008). The median PFS was 5.5 months with imlunestrant vs 3.8 months with standard endocrine therapy. The ORR among patients with measurable disease was 14.3% vs 7.7%, respectively. Most adverse effects were grade 1/2, with discontinuations in 4.6% of patients.

Clinical significance: Imlunestrant provides the first FDA-approved oral selective estrogen receptor degrader (SERD) for patients with ESR1-mutated breast cancer, a mutation that drives resistance to standard hormone therapies. This approval expands treatment options with a targeted, well-tolerated, and convenient oral therapy for a population with limited alternatives.

ALSO APPROVED: The FDA also cleared the Guardant360 CDx as a companion diagnostic to identify patients with ESR1-mutated advanced breast cancer who are eligible to receive imlunestrant.

OTHER RELATED COVERAGE

• In a prior Peer Exchange series, panelists explained how oral SERDs like imlunestrant, camasertinib, and giredestrant are demonstrating efficacy primarily in populations with ESR1 mutations and are all well-tolerated agents.
• In a prior interview, Erica L. Mayer, MD, MPH, of Dana-Farber Cancer Institute and Harvard Medical School, discussed the clinical significance of data from EMBER-3.
• EMBER-3 findings from the 2025 ASCO Annual Meeting showed that patient-reported outcomes were improved with imlunestrant vs standard endocrine therapy in patients with ER-positive/HER2-negative advanced breast cancer who experienced disease progression following endocrine therapy.
• Subgroup analyses from EMBER-3 showed that imlunestrant plus abemaciclib (Verzenio) showed consistent benefit over single-agent imlunestrant in CDK4/6 inhibitor–pretreated patients with ER-positive/HER2-negative advanced breast cancer, irrespective of biomarker status and prior CDK4/6 inhibitor type or duration.

OTHER NOTEWORTHY DECISIONS

• 9/2: The FDA approved rilzabrutinib (Wayrilz) for use in adult patients with persistent or chronic immune thrombocytopenia who have had insufficient response to a prior treatment based on findings from the phase 3 LUNA trial (NCT04562766).
• 9/2: The regulatory agency cleared denosumab-nxxp (Bildyos) and (Bilprevda), two biosimilars referencing denosumab (Prolia) and denosumab (Xgeva), respectively, for use in all indications of the reference products.
• 9/17: Two additional biosimilars, denosumab-kyqq (Bosaya) 60-mg/mL injection was approved for subcutaneous use in a single-dose prefilled syringe, as was denosumab-kyqq (Aukelso) 70 mg/mL injection for subcutaneous use in a single-dose vial as biosimilars for reference Prolia and Xgeva. The FDA granted a provisional interchangeability designation for Bosaya and Aukelso.
• 9/30: The FDA also approved denosumab-qdbe (Enoby) and denosumab-qdbe (Xtrenbo) biosimilars referencing Prolia and Xgeva, for use in all indications of the reference products.

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