The FDA approved pembrolizumab for treatment of certain patients with bacillus Calmette-Guérin-unresponsive, high-risk nonmuscle-invasive bladder cancer.
The approval applies to use of pembrolizumab (Keytruda, Merck) — an anti-PD-1 therapy — by patients with carcinoma in situ with or without papillary tumors who are not eligible for or have opted not to undergo cystectomy.
The agency based the approval on results of the multicenter, single-arm KEYNOTE-057 trial, which included 148 patients with high-risk nonmuscle-invasive bladder cancer. Most patients (64.8%; n = 96) had bacillus Calmette-Guérin (BCG)-unresponsive carcinoma in situ with or without papillary tumors.
Patients received 200 mg pembrolizumab every 3 weeks. Treatment continued for up to 24 months, or until unacceptable toxicity, persistent or recurrent high-risk disease or progressive disease.
Complete response and duration of response served as major efficacy outcomes.
Researchers reported a 41% (95% CI, 31-51) complete response rate among patients with high-risk BCG-unresponsive disease with carcinoma in situ. Median duration of response was 16.2 months (range, 0-30.4+). Nearly half (46%) of complete responses lasted at least 12 months.
The most common adverse reactions among pembrolizumab-treated patients in KEYNOTE-057 included fatigue, diarrhea, rash, pruritis, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism and nasopharyngitis.
The FDA previously granted pembrolizumab priority review for this indication.
The FDA has approved pembrolizumab for treatment of specific patients with melanoma, Merkel cell carcinoma, lung cancer, hepatocellular carcinoma, head and neck cancer, Hodgkin lymphoma, primary mediastinal large B-cell lymphoma, urothelial carcinoma, gastric cancer, esophageal cancer, cervical cancer, renal cell carcinoma and microsatellite instability-high cancer.