FDA grants priority review to Merck's sBLA for Keytruda in certain patients with high-risk, non-muscle invasive bladder cancer.

Merck Inc., announced that the FDA has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for Keytruda, Merck's anti-PD-1 therapy. The application seeks approval of Keytruda monotherapy 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 tumors who are ineligible for or have elected not to undergo cystectomy (removal of bladder).

This application for Keytruda which is based on results from the Phase II KEYNOTE-057 trial, will be discussed at the 17 December meeting of the FDA's Oncologic Drugs Advisory Committee (ODAC). Data from this trial were first presented at the European Society for Medical Oncology 2018 Congress. Merck anticipates a Prescription Drug User Fee Act (PDUFA), or target action date, in January 2020, based on priority review.

The filing was based on data from KEYNOTE-057 (NCT02625961), a Phase II , multicenter, open-label, single-arm trial in 102 patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in-situ (CIS) with or without papillary tumors who were ineligible for or had elected not to undergo cystectomy (Cohort A). In this study, BCG-unresponsive high risk NMIBC is defined as persistent disease despite adequate BCG therapy, disease recurrence after an initial tumor-free state following adequate BCG therapy, or T1 disease following a single induction course of BCG. Patients received Keytruda 200 mg every three weeks until unacceptable toxicity, persistent or recurrent high-risk NMIBC or progressive disease. Assessment of tumor status was performed every 12 weeks, and patients without disease progression could be treated for up to 24 months. The major efficacy outcome measures were complete response (as defined by negative results for cystoscopy [with transurethral resection of bladder tumor (TURBT)/biopsies as applicable], urine cytology, and computed tomography urography [CTU] imaging) and duration of response.