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FDA Approves Inlexzo

FDA Approves Inlexzo (gemcitabine intravesical system) for the Treatment of Non-Muscle Invasive Bladder Cancer

September 2025

September 9, 2025. RARITAN, N.J., September 9, 2025 /PRNewswire/ – Johnson & Johnson (NYSE:JNJ) announced today the U.S. Food and Drug Administration (FDA) approved Inlexzo (gemcitabine intravesical system), a new, potentially practice-changing approach for treating patients with certain types of bladder cancer, addressing the need for additional options following unsuccessful BCG therapy and for patients refusing or ineligible for bladder removal surgery (radical cystectomy). Inlexzo, previously referred to as TAR-200, is indicated 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.1

Inlexzo is designed for patients seeking bladder preservation and is the first and only intravesical drug releasing system (iDRS) to provide extended local delivery of a cancer medication into the bladder. Inlexzo remains in the bladder for three weeks per treatment cycle for up to 14 cycles.1 A healthcare professional places Inlexzo into the bladder using a co-packaged urinary catheter and stylet to insert it into the bladder. Inlexzo is placed in an outpatient setting in a few minutes, without the need for general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.1

“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 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.”

The approval is supported by data from the SunRISe-1 ( NCT04640623) single arm, open-label Phase 2b clinical study.1 Results show 82 percent of patients with BCG-unresponsive NMIBC treated with Inlexzo achieved a complete response (CR), meaning no signs of cancer were found after treatment (95 percent confidence interval [CI], 72, 90).1 This high response rate demonstrated strong durability, and 51 percent of these patients maintained a complete response for at least one year.1

In the SunRISe-1 clinical trial supporting this approval, the most common adverse reactions (≥15 percent) including laboratory abnormalities, 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).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, M.D., 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 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 John Reed, M.D., Ph.D., Executive Vice President, R&D, Innovative Medicine, Johnson & Johnson. “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).** “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.”

Leading to today’s approval, the FDA granted Inlexzo Breakthrough Therapy Designation (BTD), Real-Time Oncology Review (RTOR), and Priority Review.

Johnson & Johnson is committed to helping patients access our treatments. Once a patient and their doctor have decided that Inlexzo is right for the patient, J&J withMe provides a simple, comprehensive patient support program offering cost support, a dedicated Care Navigator and educational resources, at no cost to the patient.

About SunRISe-1, Cohort 2SunRISe-1 ( NCT04640623), Cohort 2, was a single arm, open-label Phase 2b clinical study that evaluated the safety and efficacy of Inlexzo monotherapy for BCG-unresponsive NMIBC patients with carcinoma in situ (CIS) with or without papillary tumors who are ineligible for, or elected not to undergo, radical cystectomy. The primary endpoint for Cohort 2 was complete response (CR) rate at any time point, and secondary endpoints include duration of response (DOR).

About Non-Muscle Invasive Bladder Cancer (NMIBC) and the Current Standard of Care Non-muscle invasive bladder cancer (NMIBC) is a type of non-invasive bladder cancer that can be classified as low, intermediate, or high risk depending on the presence of characteristics including tumor size, presence of multiple tumors, and carcinoma in situ (CIS).2 NMIBC with CIS makes up approximately 10 percent of patients with NMIBC.3 The current standard of care for NMIBC is Bacillus Calmette-Guérin (BCG), which is a weakened form of the bacteria found in tuberculosis treatment. Though effective, some patients become unresponsive to it and may experience challenges.4,5 Radical cystectomy is currently recommended for NMIBC patients who fail BCG therapy; it is a life-altering surgery with a high degree of morbidity and adverse impact on life, and has a post-surgery mortality rate of three to eight percent.6,7 Given that NMIBC typically affects older patients, many may be unwilling or unfit to undergo radical cystectomy.

About Inlexzo

Inlexzo is approved by the U.S. Food and Drug Administration (FDA) 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. Inlexzo is an intravesical system enabling extended release of gemcitabine into the bladder. It is placed in a few minutes without general anesthesia or further monitoring immediately post-insertion within the healthcare provider’s office.

The safety and efficacy of Inlexzo is being evaluated in clinical trials in patients with MIBC in SunRISe-4, and NMIBC in SunRISe-1, SunRISe-3, and SunRISe-5.

INDICATION AND IMPORTANT SAFETY INFORMATION

INDICATION

Inlexzo (gemcitabine intravesical system) is indicated 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.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

Inlexzo is contraindicated in patients with:

WARNINGS AND PRECAUTIONS

Risks in Patients with Perforated Bladder

Inlexzo may lead to systemic exposure to gemcitabine and to severe adverse reactions if administered to patients with a perforated bladder or to those in whom the integrity of the bladder mucosa has been compromised.

Evaluate the bladder before the intravesical administration of Inlexzo and do not administer to patients with a perforated bladder or mucosal compromise until bladder integrity has been restored.

Risk of Metastatic Bladder Cancer with Delayed Cystectomy

Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. The risk of developing muscle invasive or metastatic bladder cancer increases the longer cystectomy is delayed in the presence of persisting CIS.

Of the 83 evaluable patients with BCG-unresponsive CIS treated with Inlexzo in Cohort 2 of SunRISe-1, 7 patients (8%) progressed to muscle invasive (T2 or greater) bladder cancer. Three patients (3.5%) had progression determined at the time of cystectomy. The median time between determination of persistent or recurrent CIS or T1 and progression to muscle invasive disease was 94 days.

Magnetic Resonance Imaging (MRI) Safety

Inlexzo can only be safely scanned with MRI under certain conditions. Refer to section 5.3 of the USPI for details on conditions.

Embryo-Fetal Toxicity

Based on animal data and its mechanism of action, Inlexzo can cause fetal harm when administered to a pregnant woman if systemic exposure occurs. In animal reproduction studies, systemic administration of gemcitabine was teratogenic, embryotoxic, and fetotoxic in mice and rabbits.

Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment and for 6 months after final removal of Inlexzo. Advise male patients with female partners of reproductive potential to use effective contraception during treatment and for 3 months after final removal of Inlexzo.

ADVERSE REACTIONS

Serious adverse reactions occurred in 24% of patients receiving Inlexzo. Serious adverse reactions that occurred in >2% of patients included urinary tract infection, hematuria, pneumonia, and urinary tract pain. Fatal adverse reactions occurred in 1.2% of patients who received Inlexzo, including cognitive disorder.

The most common (>15%) adverse reactions, including laboratory abnormalities, were urinary frequency, urinary tract infection, dysuria, micturition urgency, decreased hemoglobin, increased lipase, urinary tract pain, decreased lymphocytes, hematuria, increased creatinine, increased potassium, increased AST, decreased sodium, bladder irritation, and increased ALT.

USE IN SPECIFIC POPULATIONS

Pregnancy

There are no available data on the use of Inlexzo in pregnant women to inform a drug-associated risk.

Please see Embryo-Fetal Toxicity for risk information related to pregnancy.

Lactation

Because of the potential for serious adverse reactions in breastfed infants, advise women not to breastfeed during treatment and for 1 week after final removal of Inlexzo.

Females and Males of Reproductive Potential

Pregnancy Testing - Verify pregnancy status in females of reproductive potential prior to initiating Inlexzo.

Contraception - Please see Embryo-Fetal Toxicity for information regarding contraception.

Infertility (Males) - Based on animal studies, Inlexzo may impair fertility in males of reproductive potential. It is not known whether these effects on fertility are reversible.

Geriatric Use

Of the patients given Inlexzo monotherapy in Cohort 2 of SunRISe-1, 72% were 65 years of age or older and 34% were 75 years or older. There were insufficient numbers of patients <65 years of age to determine if these patients respond differently to patients 65 years of age and older.

Please read the full Prescribing Information and Instructions for Use for Inlexzo.

  1. Inlexzo U.S. Prescribing Information.
  2. Grab-Heyne K, Henne C, Mariappan P, et al. Intermediate and high-risk non-muscle-invasive bladder cancer: an overview of epidemiology, burden, and unmet needs. Front Oncol. 2023;13:1170124. doi:10.3389/fonc.2023.1170124
  3. Llano A, Chan A, Kuk C, et al. Carcinoma in situ (CIS): Is there a difference in efficacy between various BCG strains? A comprehensive review of the literature. Cancers (Basel). 2024;16(2):245. doi:10.3390/cancers16020245
  4. Jiang S, Redelman-Sidi G. BCG in bladder cancer immunotherapy. Cancers (Basel). 2022;14(13):3073. doi:10.3390/cancers14133073
  5. Al Hussein Al Awamlh B, Chang SS. Novel therapies for high-risk non-muscle invasive bladder cancer. Curr Oncol Rep. 2023;25(2):83-91. doi:10.1007/s11912-022-01350-9
  6. Aminoltejari K, Black PC. Radical cystectomy: a review of techniques, developments and controversies. Transl Androl Urol. 2020;9(6):3073-3081. doi:10.21037/tau.2020.03.23
  7. Marqueen KE, Waingankar N, Sfakianos JP, et al. Early mortality in patients with muscle-invasive bladder cancer undergoing cystectomy in the United States. JNCI Cancer Spectr. 2018;2(4):pky075. doi:10.1093/jncics/pky075

Source: Johnson & Johnson

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