Image Credit: Adobe Stock Images/Mariya

The FDA has approved Johnson & Johnson’s (J&J) Inlexzo (gemcitabine intravesical system), a novel therapy for adults with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. The approval was based on results from the Phase IIb SunRISe-1 trial (NCT04640623), which demonstrated a high number of complete responses (CR).¹

How does Inlexzo Offer a New Treatment Option for Patients Unresponsive to Traditional Bladder Cancer Therapy?

“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, EVP, worldwide chairman, innovative medicine, J&J, in a press release. “In an area that has seen little progress for more than 40 years, Inlexzo delivers a first-of-its-kind breakthrough innovation with a bright future ahead.”

SunRISe-1 Trial Design and Treatment

• Cohort 2 of the single-arm, open-label SunRISe-1 trial evaluated the safety and efficacy of Inlexzo as a monotherapy in patients with BCG-unresponsive NMIBC patients with CIS, with or without papillary tumors who are ineligible for or elected not to undergo radical cystectomy.
• The treatment was administered as a three-week intravesical cycle, with up to 14 cycles, via a catheter in an outpatient setting without general anesthesia.
• The primary endpoint for cohort 2 was CR rate at any time.
• Key secondary endpoints included duration of response, number of patients with anti-cetrelimab antibodies, and the number of patients with adverse events (AEs) by severity grades.^1,2

Clinical Results

• Results showed that 82% of patients treated with Inlexzo achieved a CR, with 51% maintaining a CR for at least one year.
• The most common AEs were urinary frequency, dysuria, urinary tract infections, urgency, hematuria, bladder irritation, and laboratory abnormalities.
• Inlexzo was reported to be well tolerated.¹

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

In July, Inlexzo was granted Priority Review based on promising results from all four cohorts of the SunRISe-1 trial.³ The drug has also been granted Breakthrough Therapy Designation and Real-Time Oncology Review status.

“We are proud of the science that has brought us to this historic moment,” said John Reed, MD, PhD, EVP, R&D, innovative medicine, J&J, in the press release. “Inlexzo is a novel therapy with powerful efficacy and demonstrated safety profile. As the only major healthcare company that hosts both pharmaceuticals and medical devices, we leveraged the speed and scale of Johnson & Johnson to accelerate innovation and deliver this important therapy to patients.”

Bladder Cancer Context

• According to the American Cancer Society, bladder cancer is more common in men than in women.
• Currently, bladder cancer is the tenth leading cause of cancer-related deaths in the United States.
• Males currently have a one in 125 chance of dying from bladder cancer.
• However, incidence rates have declined by 1% per year in recent years, mainly due to a decline in smoking.⁴

“At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network, in the press release. “This approval represents the kind of progress that brings new options to a community that urgently needs them. Patients with bladder cancer need guidance and collaboration with providers to navigate bladder-sparing treatment options, including newly approved treatments like Inlexzo, so they can move forward feeling well-informed and confident.”

References

1. U.S. FDA approval of INLEXZO™ (gemcitabine intravesical system) set to transform how certain bladder cancers are treated. Johnson & Johnson. September 9, 2025. Accessed September 10, 2025. https://www.jnj.com/media-center/press-releases/u-s-fda-approval-of-inlexzo-gemcitabine-intravesical-system-set-to-transform-how-certain-bladder-cancers-are-treated
2. A Study of TAR-200 in Combination With Cetrelimab, TAR-200 Alone, or Cetrelimab Alone in Participants With Non-Muscle Invasive Bladder Cancer (NMIBC) Unresponsive to Intravesical Bacillus Calmette-Guérin Who Are Ineligible for or Elected Not to Undergo Radical Cystectomy (SunRISe-1). Clinicaltrials.gov. Accessed September 10, 2025. https://clinicaltrials.gov/study/NCT04640623
3. FDA Grants Priority Review to Johnson & Johnson’s TAR-200 for High-Risk Non-Muscle Invasive Bladder Cancer. PharmExec. July 17, 2025. Accessed September 10, 2025. https://www.pharmexec.com/view/fda-grants-priority-review-johnson-johnson-tar-200-high-risk-non-muscle-invasive-bladder-cancer
4. Key Statistics for Bladder Cancer. American Cancer Society. Accessed September 10, 2025. https://www.cancer.org/cancer/types/bladder-cancer/about/key-statistics.html