Bladder Cancer

A recap of the FDA submissions and regulatory decisions in urology from January 2025.

“Black patients have over 2-fold excess mortality compared to White men, and that has been fairly consistent; the actual rate ratio has hovered between 2 and 2.5 for many years,” says Matthew R. Cooperberg, MD, MPH.

A list of facts and trends to know about the current landscape of urologic cancer management and research in the US.

Two recent roundtable discussions focused on the challenges and evolving treatment strategies in upper tract urothelial carcinoma.

Panelists discuss how BCG-unresponsive bladder cancer is defined by disease persistence/recurrence within 6 to 12 months of adequate BCG therapy. Treatment options include cystectomy, intravesical chemotherapy, immunotherapy, or clinical trials.

Panelists discuss how low-risk non–muscle-invasive bladder cancer (NMIBC) requires transurethral resection of bladder tumor (TURBT) with surveillance. Intermediate-risk disease needs adjuvant intravesical chemotherapy. High-risk cases receive BCG induction/maintenance therapy after TURBT, with close monitoring.

Check out the key regulatory decisions set to happen early this year.

If approved, the marketing authorization application would be valid in all 27 European Union member states as well as in Iceland, Liechtenstein, and Norway.

"The three-year durability data from this study further validate the potential of JELMYTO in providing long-term disease control for patients with low-grade upper tract urothelial cancer,” says Solomon L. Woldu, MD.

The accuracy of PLND was 93.41% in the ICG injection arm compared with 75.91% in the control arm.

"At Mayo Clinic, we think that delivering intravesical therapy for non-muscle invasive bladder cancer in patients' homes has the potential to reduce treatment burden, improve the patient experience, as well as increase access to care," says Timothy D. Lyon, MD.

“If patients are not experiencing a clear clinical benefit for chemotherapy prior to surgery, we need to be really thoughtful about how we use it,” says Fed Ghali, MD.

"My biggest hope for people reading this review is to understand that there are some golden opportunities for research," says Laura Bukavina, MD, MPH, MSc.

A panelist discusses how significant unmet needs in NMIBC treatment persist around BCG supply shortages, optimal treatment sequencing, biomarker development for patient selection, and reducing treatment toxicity, while expressing optimism about emerging trends in combination therapies, novel delivery systems, and personalized medicine approaches based on molecular profiling.

The planned sBLA will seek approval of Anktiva for patients with BCG-unresponsive NMIBC in the papillary indication.

The NDA is supported by data from cohort 2 of the phase 2b SunRISe-1 trial.

"I think integrating microbiome surveillance into clinical trials can bridge the gap between research and also therapeutic applications," says Ilaha Isali, MD, MSc.

“In this phase 2 study, an image-guided adaptive strategy enabled radiotherapy dose escalation to over 86% of patients’ bladder tumors without significant increase in toxicity,” wrote the authors.

“It's hard for us, based on our evidence here, to say that neoadjuvant chemotherapy is causal in any way or is resulting in prolonged survival,” says Fed Ghali, MD.

"The increasing expenses associated with bladder cancer treatment highlight wider issues regarding the sustainability of health care expenditures in the US," writes Michael S. Cookson, MD, MMHC, FACS.

Results from CREST will be presented at an upcoming medical conference.

"We expect to initiate patient dosing in the second quarter of this year, with initial three-month [complete response] data to follow," says Doug Warner.

A panelist discusses how emerging investigational agents for NMIBC show promise through diverse mechanisms, including oncolytic viruses (CG0070), immune checkpoint inhibitors (durvalumab, sasanlimab), targeted drug delivery systems (TAR-200), mitomycin-containing gels (UGN-102/103), immunotherapies (TARA-002), gene therapies (EG-70), and FGFR inhibitors (erdafitinib), representing a robust pipeline of potential treatment options.

9MW2821 was previously awarded a breakthrough therapy designation as a monotherapy for locally advanced or metastatic urothelial carcinoma.

"Studying the microbiome overall is challenging because it's dirty science. You have to be incredibly careful with contamination," says Ilaha Isali, MD, MSc, and Laura Bukavina, MD, MPH, MSc.

At a median follow-up of 33.2 months, the confirmed objective response rate was 73.2%.

The NMPA’s approval was supported by data from the phase 3 EV-302 trial.

"The interplay between the microbiome and bladder cancer is very complex," says Ilaha Isali, MD, MSc.

“It's a good problem to have, because we're thinking about a previous era in which we didn't have many options besides more BCG vs cystectomy,” says Vikram M. Narayan, MD.

The J-code became effective on January 1, 2025.