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

Efficacy was evaluated in KEYNOTE-716 (NCT03553846), a multicentre, randomised (1:1), double-blind, placebo-controlled study in patients with completely resected stage IIB or IIC melanoma. Patients were randomised to pembrolizumab 200 mg or the paediatric (≥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 study demonstrated a statistically significant improvement in RFS at the time of the first interim analysis for patients randomised to the pembrolizumab arm compared with placebo, with a hazard ratio of 0.65 (95% confidence interval 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, diarrhoea, pruritus, and arthralgia.

Full prescribing information for Keytruda is available here.

This review used the Real-Time Oncology Review pilot programme, 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.

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. 

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA’s Oncology Center of Excellence Project Facilitate