December 5, 2016

The Food and Drug Administration (FDA) approved nivolumab (Opdivo®) on November 10 for the treatment of squamous cell cancer of the head and neck (SCCHN).

Nivolumab is already approved for the treatment of several other cancers. This new approval is for the use of nivolumab in patients with SCCHN that has progressed during chemotherapy with a platinum-based drug or that has recurred or metastasized after platinum-based chemotherapy.

Nivolumab is the second immunotherapy drug approved to treat SCCHN. In August of this year, the FDA approved pembrolizumab (Keytruda®) for patients with SCCHN whose disease has progressed during or after platinum-containing chemotherapy. Both nivolumab and pembrolizumab are immune checkpoint inhibitors, drugs that prevent tumor cells from blocking attack by the immune system.

Previously, nivolumab has been approved to treat certain types of Hodgkin lymphoma, renal cell carcinoma, non-small cell lung cancer, and melanoma.

The approval for SCCHN was based on findings from a 361-patient clinical trial, funded by Bristol-Myers Squibb, in which participants were randomly assigned to receive either nivolumab or one of three standard therapies: cetuximab (Erbitux®), , or docetaxel.

Patients in the nivolumab group lived a median of 7.5 months while patients in the standard therapy group lived a median of 5.1 months. In addition, more patients in the nivolumab group had their tumors shrink or disappear during treatment: 6 patients had a complete response, and 26 had a partial response. In the standard therapy group, only 1 had a complete response and 6 had a partial response.

“Until this point, there were not a lot of helpful options available for patients, outside of cetuximab or the platinum-containing agents, if their cancer had progressed after first-line therapy,” said Sharon Spencer, M.D., of the University of Alabama at Birmingham Comprehensive Cancer Center and vice-chair of the National Comprehensive Cancer Network’s head and neck cancer treatment guidelines panel.

The immune checkpoint inhibitors “are a great new category of drug that’s now available, and the rapid [tumor] regressions that some patients experience are very exciting,” Dr. Spencer added.

In the clinical trial, 13.1% of patients taking nivolumab had severe side effects related to treatment compared with 35.1% of patients in the standard therapy group. Cough and trouble breathing (dyspnea) were the most common serious side effects in patients treated with nivolumab.

During the trial, two patients in the nivolumab group and one patient in the standard therapy group died from treatment-related side effects. (Twice as many patients in the trial received nivolumab than did standard therapy.)

Patients treated with nivolumab reported having better quality of life during treatment—based on measures such as pain and physical functioning—than patients treated with standard therapy. Improvements in symptoms for patients with recurrent advanced head and neck cancer is essential to an improved quality of life, explained Dr. Spencer.

Immune checkpoint inhibitors “can often decrease symptoms for patients with head and neck cancer who have large tumor burdens. These symptoms are really difficult to control,” she said. “So anything that lessens the tumor burden is a win for them.”