Oops, you're using an old version of your browser so some of the features on this page may not be displaying properly.

MINIMAL Requirements: Google Chrome 24+Mozilla Firefox 20+Internet Explorer 11Opera 15–18Apple Safari 7SeaMonkey 2.15-2.23

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

Efficacy was evaluated in KEYNOTE-057 trial
09 Jan 2020
Cancer Immunology and Immunotherapy;  Genitourinary cancers

On 8 January 2020, the US Food and Drug Administration (FDA) approved pembrolizumab (KEYTRUDA, Merck & Co. Inc.) for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumours who are ineligible for or have elected not to undergo cystectomy.   

Efficacy was investigated in KEYNOTE-057, a multicenter, single-arm trial that enrolled 148 patients with high-risk NMIBC, 96 of whom had BCG-unresponsive CIS with or without papillary tumours. Patients received pembrolizumab 200 mg every 3 weeks until unacceptable toxicity, persistent or recurrent high-risk NMIBC or progressive disease, or up to 24 months of therapy without disease progression. 

The major efficacy outcome measures were complete response (as defined by negative results for cystoscopy [with TURBT/biopsies as applicable], urine cytology, and computed tomography urography imaging) and duration of response. The complete response rate in the 96 patients with high-risk BCG-unresponsive NMIBC with CIS was 41% (95% CI: 31, 51) and median response duration was 16.2 months (0.0+, 30.4+). In total, 46% of responding patients experienced a complete response lasting at least 12 months. 

The most common adverse reactions (incidence ≥10%) in patients who received pembrolizumab in KEYNOTE-057 were fatigue, diarrhoea, rash, pruritis, musculoskeletal pain, haematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral oedema, hypothyroidism, and nasopharyngitis.

The recommended pembrolizumab dose is 200 mg every 3 weeks. 

Full prescribing information for KEYTRUDA is available here.  

Pembrolizumab was granted priority review.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System. 

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA Oncology Center of Excellence’s Project Facilitate.

This site uses cookies. Some of these cookies are essential, while others help us improve your experience by providing insights into how the site is being used.

For more detailed information on the cookies we use, please check our Privacy Policy.

Customise settings