Johnson & Johnsonannounced that the U.S. Food and Drug Administration (FDA) has approved INLEXZO™ (gemcitabine intravesical system), marking a potentially transformative advancement in the treatment of certain types of bladder cancer. This novel medicine is indicated for adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), a high-risk form of the disease. INLEXZO™ offers a crucial new option for patients who have not responded to standard BCG therapy and for those who are either ineligible for or choose to avoid radical cystectomy, a life-altering bladder removal surgery that is currently the recommended course of action after BCG failure. The approval is a significant breakthrough, as there has been little progress in this area for more than 40 years.
INLEXZO™ is the first and only intravesical drug releasing system (iDRS) designed to provide extended, local delivery of a cancer medication directly into the bladder.
The system is placed by a healthcare professional in an outpatient setting in just a few minutes, without the need for general anesthesia or immediate post-insertion monitoring. Each treatment cycle involves the system remaining in the bladder for three weeks, and patients can receive up to 14 cycles. This innovative approach offers a bladder-preserving alternative to surgery, addressing a major unmet need for patients, particularly older individuals who may not be able to tolerate a major operation.
The FDA approval was supported by compelling data from the SunRISe-1 Phase 2b clinical study. Results from the single-arm trial showed an impressive 82 percent complete response rate, meaning no signs of cancer were detected after treatment. Furthermore, the study demonstrated strong durability, with 51 percent of these patients maintaining their complete response for at least one year. While the treatment was clinically meaningful, the study noted common adverse reactions, including urinary frequency, urinary tract infection, pain, and various laboratory abnormalities.
The approval was accelerated through FDA designations including Breakthrough Therapy, Real-Time Oncology Review, and Priority Review, underscoring the therapy's importance.
A principal investigator for the study, Dr. Sia Daneshmand, praised the therapy as a well-tolerated option that delivers clinically meaningful results and will likely change the way bladder cancer is treated. Johnson & Johnson has also committed to patient access by offering the J&J withMe support program, which provides cost support and educational resources.