FDA approved a new drug, Zelboraf to treat advanced form of skin cancer (late-stage melanoma). Late-stage melanoma is the most most dangerous type of skin cancer, after it was shown to extend patients' survival.
Made by Genentech, a US subsidiary of the Swiss pharmaceutical giant Roche, Zelboraf (vemurafenib) is the second melanoma drug this year, following Yervoy (ipilimumab) in March, and was given a rush approval by US regulators.
AdvertisementThe treatment only works in about half of patients with advanced melanoma, those whose tumors express a gene mutation called BRAF V600E, meaning it could help about 10,000 patients in the United States, according to experts.
Just a few treatments for melanoma currently exist, with little success in extending the life of patients. Most people diagnosed with advanced melanoma die within 11 months, said Tim Turnham of the Melanoma Research Foundation.
"This is a really big deal," Turnham told AFP. "This is two drugs after 13 years of nothing."
Zelboraf works by blocking a protein that is involved with cell growth.
"This is a whole new approach to tackling melanoma," explained Turnham. "This actually goes into the malignant tumor cells and shuts them down."
The FDA said the approval of Zelboraf comes with a "first-of-a-kind test" called the cobas 4800 BRAF V600 mutation test to determine if patients have the type of cancer that the drug can treat.
"Today's approval of Zelboraf and the cobas test is a great example of how companion diagnostics can be developed and used to ensure patients are exposed to highly effective, more personalized therapies in a safe manner," said Alberto Gutierrez, director of the Office of In Vitro Diagnostic Device Evaluation and Safety in the FDA's Center for Devices and Radiological Health.
The regulatory agency had set a goal date of deciding on the drug by late October, but issued the decision early after a promising results from an international trial of 675 patients with late-stage melanoma with the BRAF V600E mutation.
The FDA said that compared to another anti-cancer therapy, dacarbazine, Zelboraf showed longer overall survival, or the length of time between the start of treatment and the patient's death.
"The median survival (the length of time a patient lives after treatment) of patients receiving Zelboraf has not been reached (77 percent still living) while the median survival for those who received dacarbazine was eight months (64 percent still living)," FDA said.
Early findings from that phase-three clinical trial were presented in Chicago at June's conference of the American Society of Clinical Oncology (ASCO).
Lead author Paul Chapman, a physician at Memorial Sloan-Kettering Cancer Center in New York, called Zelboraf "the first successful melanoma treatment tailored to patients who carry a specific gene mutation in their tumor."
Side effects include joint pain, rash, hair loss, fatigue, nausea, and sensitivity to sun exposure, and those taking it should stay out of the sun, the FDA said.
Just over a quarter of patients on Zelboraf developed another form of skin cancer, cutaneous squamous cell carcinoma, which was managed with surgery.
However, Turnham noted that while the drug can work "like magic" for some patients, its effects do not typically last.
"For a lot of people, it works like magic. Two weeks after taking Zelboraf, the cancer is gone. It's amazing the way it melts tumors away.
"But the median response time is six months, then the tumors start coming back. There is a real need to find ways to extend that response time, perhaps by combining it with other drugs," he said.
The trial of Yervoy, approved by the FDA in March, showed that the drug taken in combination with chemotherapy also prolonged the life of people with advanced melanoma compared with chemotherapy alone.
Survival rate at three years was 20.8 percent for patients with Yervoy plus chemotherapy, against 12.2 percent without Yervoy.
According to the National Cancer Institute, 68,130 new cases of melanoma were diagnosed in the United States last year and about 8,700 people died from the disease.
According to the World Health Organization, skin cancer leads to 66,000 deaths annually worldwide, 80 percent of which involve melanomas.
More than half the patients are under age 59.
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