FDA approves pembrolizumab for adjuvant treatment of Stage IIB or IIC melanoma

On December 3, 2021, the Food and Drug Administration approved pembrolizumab (Keytruda, Merck) for the adjuvant treatment of adult and pediatric (≥12 years of age) patients with stage IIB or IIC melanoma following complete resection. 

Efficacy was evaluated in KEYNOTE-716 (NCT03553836), a multicenter, randomized (1:1), double-blind, placebo-controlled trial in patients with completely resected stage IIB or IIC melanoma. Patients were randomized to pembrolizumab 200 mg or the pediatric (≥12 years) dose of 2 mg/kg intravenously (up to a maximum of 200 mg) every three weeks or placebo for up to one year until disease recurrence or unacceptable toxicity. The major efficacy outcome measure was investigator-assessed recurrence-free survival (RFS). The trial demonstrated a statistically significant improvement in RFS at the time of the first interim analysis for patients randomized to the pembrolizumab arm compared with placebo, with a hazard ratio of 0.65 (95% CI: 0.46, 0.92; p=0.0132). The median RFS was not reached in either arm. 

The most common adverse reactions reported in KEYNOTE-716 (≥20%) were fatigue, diarrhea, pruritus, and arthralgia.  

View full prescribing information for Keytruda. 

This review used the Real-Time Oncology Review (RTOR) 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. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. 

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 or by calling 1-800-FDA-1088. 

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.