FDA Approves Intravesical Gemcitabine Delivery for Bladder Cancer

The FDA has approved the intravesical gemcitabine delivery system Inlexzo, previously known as TAR-200, for the treatment of certain types of bladder cancer following unsuccessful Bacillus Calmette-Guérin (BCG) therapy or for patients who refuse/are ineligible for radical cystectomy.¹

“I see many patients that ultimately become BCG-unresponsive and often face life-altering bladder removal. These patients now may be ideal candidates for newly approved Inlexzo,” said Sia Daneshmand, MD, TAR-200 SunRISe-1 principal investigator, and professor of urology, Director of Urologic Oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California, in a press release. “In my experience, [TAR-200] is well-tolerated and delivers clinically meaningful results. This will change the way we treat appropriate patients that haven't responded to traditional therapy.”

Data from the phase 2b SunRISe-1 study (NCT04640623) support this approval. Here, 82% of patients with BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC) with or without carcinoma in situ (CIS) treated with TAR-200 achieved a complete response (CR; 95% CI, 72%–90%). Further, 51% of patients maintained a CR for at least 1 year.

Data presented at the 2025 American Urological Association (AUA) Annual Meeting also showed that the median onset to CR was 2.8 months (range, 2.1–8.3), with 95.7% (n = 67/70) achieving a CR within the first 3 months.² The Kaplan-Meier–estimated 12- and 24-month CR rates were 52.4% (95% CI, 40.7%–62.8%) and 44.7% (95% CI, 33.1%–55.7%), respectively.

Regarding safety, the most common (≥15%) adverse effects observed in SunRISe-1 were urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased aspartate aminotransferase (AST), decreased sodium, bladder irritation, and increased alanine transaminase (ALT).

“When we acquired this novel therapy in 2019, our ambition was to give patients with bladder cancer a renewed sense of hope and belief,” said Jennifer Taubert, executive vice president, worldwide chairman, Innovative Medicine, Johnson & Johnson, in the press release. "In an area that has seen little progress for more than 40 years, [TAR-200] delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”

REFERENCES:

1. U.S. FDA approval of INLEXZO™ (gemcitabine intravesical system) set to transform how certain bladder cancers are treated. News Release. Johnson & Johnson. September 9, 2025. Accessed September 9, 2025. https://tinyurl.com/yc48x4bn

2. Jacob JM, Guerrero-Ramos F, Necchi A, et al. TAR-200 monotherapy in patients with Bacillus Calmette-Guerin–unresponsive high-risk non–muscle-invasive bladder cancer carcinoma in situ: 1-year durability and patient-reported outcomes from SunRISe-1. Presented at: American Urological Association Annual Congress; April 26-29, 2025; Las Vegas, NV. P2s.