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FDA Approves First Immunotherapy Treatment for Lung Cancer

, by NCI Staff

On March 4, the U.S. Food and Drug Administration approved nivolumab (Opdivo) to treat patients with advanced non-small cell lung cancer (NSCLC) that has progressed during or after treatment with platinum-based chemotherapy.

Nivolumab, which was initially approved for the treatment of metastatic melanoma, is the first immunotherapy drug to be approved to treat lung cancer. It works by inhibiting a protein receptor called PD-1 on T cells, a type of immune cell.

PD-1 belongs to a family of so-called checkpoint proteins that, when activated, serve as a brake on the immune system. Nivolumab prevents tumor cells from communicating through the PD-1 protein to inactivate T cells, allowing the immune system to attack the tumor cells.

The FDA based the approval on findings from a randomized phase III trial that enrolled 272 patients with advanced non-small cell lung cancer who were assigned to receive either nivolumab or the chemotherapy drug docetaxel.

Results from the phase III trial have yet to be published or presented at a scientific meeting, but, according to the FDA, participants who received nivolumab had a 41% reduction in the risk of death and lived on average 3.2 months longer than those who received docetaxel. Approximately 30 percent of patients treated with nivolumab were alive 2 years after beginning treatment compared to 13 percent of patients treated with docetaxel.

The FDA also cited safety and efficacy data from a phase II trial that included 117 participants whose cancer had progressed after they had received a platinum-based chemotherapy and at least one other prior systemic treatment. According to updated results from the trial (unpublished but available in the nivolumab package insert), 17 participants (14.5 percent) experienced a reduction in tumor size, of whom 59 percent had response durations of 6 months or longer. Median overall survival was 8.2 months and overall survival at 1 year was 40.8 percent.

The most common side effects in the clinical trials were fatigue, shortness of breath, musculoskeletal pain, decreased appetite, cough, nausea, and constipation.

“The fact that we now have evidence of efficacy in the most common lethal tumor, non-small cell lung cancer, solidly cements immunotherapy as a critical tool in our fight against cancer,” said James Gulley, M.D., director of the Medical Oncology Service in the NCI Center for Cancer Research.

“The rapid and durable responses seen with immune checkpoint inhibitors is a unique aspect of their clinical activity,” Dr. Gulley added. Researchers are already testing combining immune checkpoint inhibitors with other therapeutic modalities “to further increase the proportion of patients who respond,” he said.

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