MSD’s blockbuster cancer drug Keytruda (pembrolizumab) has won approval from the US Food and Drug Administration (FDA) to treat a type of head and neck cancer.

It was approved in the Indication of first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma as monotherapy for patients whose tumours express PD-L1 or in combination with platinum and fluorouracil (FU) regardless of PD-L1 expression.

The approval is based on results from the pivotal Phase 3 KEYNOTE-048 trial, where Keytruda demonstrated a significant improvement in overall survival (OS) compared with the current standard of care, the EXTREME regimen which consists of cetuximab with carboplatin or cisplatin plus FU.

The results mean that the drug is the first anti-PD-1 therapy approved in the first-line setting as monotherapy in patients whose tumours express PD-L1, and also  the first anti-PD-1 therapy to demonstrate a statistically significant improvement in OS in these patients.

The approval marks a “very exciting milestone in the treatment of head and neck cancer and has the potential to transform the way we treat patients with this debilitating disease by offering important new therapeutic options,” said Dr. Barbara Burtness, professor of medicine.

She continued, “Metastatic or recurrent head and neck cancer has been an area of significant unmet need, so it is encouraging to have immunotherapy regimens available for patients in the first-line setting.”

Keytruda was initially approved for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy in 2016 under the FDA’s accelerated approval process.