New Pembrolizumab Indication Granted FDA Approval

Officials with the FDA have approved pembrolizumab (Keytruda, Merck) 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, according to the FDA.¹

The drug is a programmed death receptor-1 (PD-1)-blocking antibody. Granted priority review for this new use, pembrolizumab also is indicated for many other types of cancer conditions.¹

The recommended pembrolizumab dose is 200 mg every 3 weeks for BCG-unresponsive, high-risk NMIBC with CIS with or without papillary.²

Efficacy for this new indication 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 tumors. 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.¹

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 [CTU] 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+). Forty-six percent of responding patients experienced a complete response lasting at least 12 months.¹

The most common adverse reactions (incidence ≥10%) in patients who received pembrolizumab in KEYNOTE-057 were fatigue, diarrhea, rash, pruritis, musculoskeletal pain, hematuria, cough, arthralgia, nausea, constipation, urinary tract infection, peripheral edema, hypothyroidism, and nasopharyngitis.¹

REFERENCES

• FDA approves pembrolizumab for BCG-unresponsive, high-risk non-muscle invasive bladder cancer [news release]. Silver Spring, MD; January 8, 2020: FDA website. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-bcg-unresponsive-high-risk-non-muscle-invasive-bladder-cancer. Accessed January 8, 2020.
• Merck. Prescribing information. FDA website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/125514s066lbl.pdf. Revised January 2020. Accessed January 8, 2020.