Abstract
The FDA approved pembrolizumab, the first PD-1 inhibitor, as a second- and third-line treatment for melanoma.
The FDA's accelerated approval of pembrolizumab (Keytruda; Merck) on September 4 will likely make a swift difference in melanoma care, according to several experts.
The drug, which turns off the immune modulator PD-1, was approved for use as a second- or third-line therapy, after the immune therapy ipilimumab (Yervoy; Bristol-Myers Squibb) no longer works, and, for BRAF V600-mutation–positive patients, after developing resistance to a BRAF inhibitor.
Pembrolizumab shows a 24% response rate and causes fewer serious side effects than ipilimumab, and yields durable results in most patients who respond, says Jeffrey Weber, MD, PhD, a senior member and director of the comprehensive melanoma research center at the H. Lee Moffitt Cancer Center in Tampa, FL, who has led studies of pembrolizumab.
“It's a major advance,” Weber says. The drug offers “great benefit for patients, humongous potential for combinations with other immunotherapies and other therapies, and it's not very toxic—you can safely treat people in their 80s.”
Weber says his experience with pembrolizumab has been positive, and he predicts that the drug will soon be approved as a first-line therapy as well. However, he does not believe that oncologists will try pembrolizumab for first-line treatment or in other types of cancer until the FDA expands its use.
Merck's PD-1 inhibitor pembrolizumab received FDA approval for the treatment of melanoma (above) in September. A similar drug from Bristol-Myers Squibb, nivolumab, may soon follow suit.
Merck's PD-1 inhibitor pembrolizumab received FDA approval for the treatment of melanoma (above) in September. A similar drug from Bristol-Myers Squibb, nivolumab, may soon follow suit.
“I don't think you're going to see a lot of off-label use of such an expensive drug,” Weber says. Estimates suggest the drug will cost $125,000 a year.
Industry experts expect a second anti–PD-1 drug, nivolumab (Bristol-Myers Squibb), to follow pembrolizumab to market in the United States in a few months.
Bristol-Myers Squibb and its partner, Ono Pharmaceutical, filed suit in federal court against Merck the same day pembrolizumab gained FDA approval, claiming Merck violated its patents. Merck denies the charge and says it expects to win the challenge.
“Getting out of the gate first is a massive advantage” for Merck, says Rachel Webster, MSc, PhD, senior director of oncology with Decision Resources Group, a health care information company headquartered in Burlington, MA, which surveys doctors.
Oncologists are well informed about pembrolizumab and will be quick to use it, Webster explains. Once they get used to prescribing it, some might be less inclined to switch to nivolumab.
The real competitive advantage, Webster says, will come from winning approval in the first-line, or treatment-naïve, setting, as well as in other cancers, such as non–small cell lung cancer and squamous cell carcinoma of the head and neck.
“The race,” she says, “has only just begun.”
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