The U.S. Food and Drug Administration today granted accelerated approval to Opdivo (nivolumab), a new treatment for patients with unresectable (cannot be removed by surgery) or metastatic (advanced) melanoma who no longer respond to other drugs.Melanoma
is the fifth most common type of cancer in the United States. It forms
in the body’s melanocyte cells, which develop the skin’s pigment. The
National Cancer Institute estimates that 76,100 Americans will be
diagnosed with melanoma and 9,710 will die from the disease this year.
Opdivo
works by inhibiting the PD-1 protein on cells, which blocks the body’s
immune system from attacking melanoma tumors. Opdivo is intended for
patients who have been previously treated with ipilimumab and, for
melanoma patients whose tumors express a gene mutation called BRAF V600, 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, M.D., director of the Office of Hematology and Oncology
Products in the FDA’s Center for Drug Evaluation and Research. “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 (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.
The FDA
granted Opvido breakthrough therapy designation, priority review and
orphan product designation because the sponsor demonstrated through
preliminary clinical evidence that the drug may offer a substantial
improvement over available therapies; the drug had the potential, at the
time of the application was submitted, to be a significant improvement
in safety or effectiveness in the treatment of a serious condition; and
the drug is intended to treat a rare disease, respectively.
Opvido
is being 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 120 clinical trial
participants with unresectable or metastatic melanoma. Results showed
that 32 percent of participants receiving Opdivo had their tumors shrink
(objective response rate). This effect lasted for more than six months
in approximately one-third of the participants who experienced tumor
shrinkage.
Opdivo’s safety was evaluated in the
overall trial population of 268 participants treated with Opdivo and 102
participants treated with chemotherapy. The most common side effects of
the drug were rash, itching, cough, upper respiratory tract infections,
and fluid retention (edema). The most serious side effects are severe
immune-mediated side effects involving healthy organs, including the
lung, colon, liver, kidneys and hormone-producing glands.
Opdivo is marketed by Princeton, New Jersey-based Bristol-Myers Squibb.
The
FDA, an agency within the U.S. Department of Health and Human Services,
promotes and protects the public health by, among other
things, assuring the safety, effectiveness, and security of human and
veterinary drugs, vaccines and other biological products for human use,
and medical devices. The agency also is responsible for the safety and
security of our nation’s food supply, cosmetics, dietary supplements,
products that give off electronic radiation, and for regulating tobacco
products.