On December 3, the U.S. Food and Drug Administration (FDA) approved pembrolizumab (Keytruda) for the adjuvant treatment of adult and pediatric patients aged 12 years and older with stage IIB or IIC melanoma following complete resection.
Efficacy was evaluated in KEYNOTE-716, a multicenter, randomized, double-blind, placebo-controlled trial in patients with completely resected stage IIB or IIC melanoma. Patients were randomly assigned to receive pembrolizumab at 200 mg or the pediatric dose of 2 mg/kg intravenously (up to a maximum of 200 mg) every 3 weeks or placebo for up to 1 year until disease recurrence or unacceptable toxicity. The major efficacy outcome measure was investigator-assessed recurrence-free survival.
The trial demonstrated a statistically significant improvement in recurrence-free survival at the time of the first interim analysis for patients randomly assigned to the pembrolizumab arm compared with placebo, with a hazard ratio of 0.65 (95% confidence interval = 0.46–0.92, P = .0132). The median recurrence-free survival was not reached in either arm.
The most common adverse reactions reported in KEYNOTE-716 (≥ 20%) were fatigue, diarrhea, pruritus, and arthralgia.
This review used the Real-Time Oncology Review pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. This application was granted Priority Review and Orphan Designation.