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The Food and Drug Administration has granted accelerated approval to nivolumab (Opdivo, made by Bristol-Myers Squibb) for use in patients with unresectable or metastatic melanoma who no longer respond to other drugs.

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Opdivo works by inhibiting the PD-1 protein on cells, which blocks the immune system from attacking melanoma cells. The drug is intended for patients who have been previously treated with ipilimumab and, for melanoma patients whose tumors express the BRAF V600 gene mutation, for use after treatment with ipilimumab and a BRAF inhibitor.


"Opdivo is the seventh new melanoma drug approved by the FDA since 2011," said Richard Pazdur, MD, Director of the FDA's Office of Hematology and Oncology Products. "The continued development and approval of novel therapies based on our increasing understanding of tumor immunology and molecular pathways are changing the treatment paradigm for serious and life-threatening diseases."


Other FDA-approved treatments for melanoma include ipilimumab (approved in 2011), peginterferon alfa-2b (2011), vemurafenib (2011), dabrafenib (2013), trametinib (2013), and pembrolizumab (2014). Opdivo is being approved more than three months ahead of the prescription drug user fee goal date of March 30, 2015, the date when the agency was scheduled to complete its review of the application.


Opdivo, which had been given both Breakthrough Therapy and Orphan Product designation, was approved under the FDA's accelerated approval program, which allows approval of a drug to treat a serious or life-threatening disease based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical trials to confirm the drug's benefit.


Opdivo's efficacy was demonstrated in a trial of 120 patients with unresectable or metastatic melanoma, and 32 percent had an objective response. The effect lasted for more than six months in approximately one-third of the participants who had tumor shrinkage.


Safety was evaluated in the overall trial population of 268 participants treated with Opdivo and 102 treated with chemotherapy. The most common side effects of Opdivo were rash, itching, cough, upper respiratory tract infections, and edema. The most serious side effects were severe immune-mediated side effects involving healthy organs, including the lung, colon, liver, kidneys, and hormone-producing glands.