FDA Approves Keytruda for BCG-Unresponsive High-Risk Non-Muscle Invasive Bladder Cancer

Today, the U.S. FDA announced the approval of Merck Sharp & Dohme’s (MSD) blockbuster PD-1 inhibitor Keytruda for the treatment of patients with specific high-risk non-muscle-invasive bladder cancer (NMIBC). These patients have carcinoma in situ (CIS), are unresponsive to Bacillus Calmette-Guérin (BCG) therapy, and are unwilling or unable to undergo cystectomy. According to MSD’s press release, this is the first PD-1 inhibitor approved for the treatment of this patient population.

Globally, approximately 430,000 people are diagnosed with bladder cancer each year, making it the ninth most common cancer worldwide. In patients with non-muscle-invasive bladder cancer (NMIBC), cancerous cells are confined to the inner lining of the bladder or have grown into the bladder lumen but have not yet spread to the muscle layer or other tissues. Approximately 75% of bladder cancer patients are diagnosed with NMIBC. Bacillus Calmette-Guérin (BCG) is the standard treatment for these patients. However, if BCG therapy fails, clinical guidelines recommend surgical removal of the entire bladder and surrounding tissues. In men, this often includes removal of the prostate, while in women, the surgery may involve removal of the uterus, fallopian tubes, ovaries, and cervix. This has a significant impact on patients’ quality of life, highlighting an urgent need for innovative treatment options beyond surgery.

Keytruda is a blockbuster PD-1 inhibitor developed by Merck & Co., Inc. (MSD). It has been approved for more than 20 indications, including the treatment of patients with advanced or metastatic urothelial carcinoma. Expanding the use of Keytruda to patients with early-stage cancer is one of MSD’s key R&D strategies. The application of Keytruda in the treatment of non-muscle-invasive bladder cancer (NMIBC) exemplifies this strategy. The regulatory application for Keytruda in NMIBC has also received Priority Review designation from the U.S. Food and Drug Administration (FDA).

This approval is based on the results of the multicenter, single-arm clinical trial named KEYNOTE-057. The trial included 148 patients with high-risk non-muscle-invasive bladder cancer (NMIBC), 96 of whom had carcinoma in situ (CIS) that was unresponsive to BCG. The trial results showed that among the 96 high-risk patients who were unresponsive to BCG, Keytruda achieved a complete response rate of 41% (95% CI: 31, 51). Among those who achieved a complete response, the median duration of response was 16.2 months. Forty-six percent of patients maintained a complete response for more than 12 months.

Recent Breakthroughs in the Treatment of NMIBC: FerGene’s Gene Therapy Meets Primary Endpoint in Phase 3 Trial for High-Risk NMIBC Patients; ImmunityBio’s IL-15 Superagonist Receives FDA Breakthrough Therapy Designation for High-Grade NMIBC Carcinoma In Situ. We look forward to the early approval of more innovative therapies, providing this patient population with non-surgical treatment options.

References:

[1] FDA approves pembrolizumab for BCG-unresponsive, high-risk non-muscle invasive bladder cancer. Retrieved January 8, 2020, from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-bcg-unresponsive-high-risk-non-muscle-invasive-bladder-cancer

Source: WuXi AppTec