FDA Approves Inlexzo for Some With Non–Muscle Invasive Bladder Cancer

The FDA has approved Inlexzo for adults with BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ.

The U.S. Food and Drug Administration (FDA) has approved Inlexzo (gemcitabine intravesical system) 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 agency announced, noting that the system is co-packaged with a urinary catheter and stylet used for insertion through the urinary catheter into the bladder.

The treatment’s effectiveness was determined in cohort 2 of the SunRISe-1 trial, which enrolled 83 patients with BCG-unresponsive NMIBC with CIS with or without papillary tumors following transurethral resection, with patients receiving Inlexzo into the bladder every three weeks for six months, followed by once every 12 weeks for up to 18 months.

The system delivers 225 mg of gemcitabine chemotherapy into the bladder, with an indwelling period following each insertion of three weeks prior to removal, and the system is inserted once every three weeks for up to six months, or eight doses, and then once every 12 weeks for up to 18 months, or six doses), or until persistent or recurrent high-grade NMIBC, disease progression or unacceptable toxicity.

The treatment, previously known as TAR-200, was granted priority review by the FDA in July.

Tumor status, as stated in the FDA’s announcement, was assessed via cystoscopy and urine cytology every 12 weeks during the initial two years of treatment, after which cystoscopy was performed at least every 24 weeks, and biopsies were performed at 24 and 48 weeks after treatment initiation.

Treatment was associated with a complete response rate of 82% and 51% of patients with a complete response had a duration of response of at least one year.

The treatment’s prescribing information, the FDA noted, includes warnings and precautions describing the risks of administering gemcitabine intravesical system in patients with a perforated bladder, risk of metastatic bladder cancer with delayed cystectomy, magnetic resonance imaging (MRI) safety and embryo-fetal toxicity.

Reactions to the FDA Approval

“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 Dr. Sia Daneshmand, principal investigator of the clinical trial and professor of urology and director of urologic oncology at the Norris Comprehensive Cancer Center, Keck School of Medicine of University of Southern California, in a statement included in a news release from Johnson & Johnson. “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.”

“We are proud of the science that has brought us to this historic moment,” said Dr. John Reed, executive vice president of R&D, Innovative Medicineat Johnson & Johnson, in the news 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."

“At BCAN, our mission has always been to advocate for better todays and more tomorrows for everyone impacted by bladder cancer. This approval represents the kind of progress that brings new options to a community that urgently needs them,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network (BCAN), in the news release. “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.”

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References:

1. “FDA approves gemcitabine intravesical system for non-muscle invasive bladder cancer,” FDA; https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gemcitabine-intravesical-system-non-muscle-invasive-bladder-cancer?check_logged_in=1
2. “U.S. FDA approval of INLEXZO™ (gemcitabine intravesical system) set to transform how certain bladder cancers are treated,” news release, Sept. 9, 2025; https://www.multivu.com/johnson-and-johnson/9342851-en-johnson-and-johnson-fda-approval-inlexzo-gemcitabine-intravesical-system