The FDA has approved Zelboraf, known as vemurafenib or PLX4032 during testing, well ahead of the approval deadline. Zelboraf is the first drug to treat advanced melanoma by targeting a specific gene mutation.
Healthcare professionals have used targeted therapies to block the growth and spread of other cancers, including breast cancer, pancreatic cancer and non-small cell lung cancer. Zelboraf is the first approved drug to use this approach to treat melanoma. The FDA took quick action on this therapy, beating their approval deadline by six months.
"We have seen evidence that targeting gene mutations works. But it also demonstrates the very real challenges researchers still face in offering patients lasting results," said Timothy J. Turnham, the executive director of the Melanoma Research Foundation (MRF).
Zelboraf is designed to kill cancer cells by blocking the mutated BRAF gene found in 40 to 60% of all melanoma patients. One trial found 84% of the advanced melanoma patients taking Zelboraf survived for at least six months, compared to 64% of the patients taking a traditional chemotherapy drug called dacarbazine. Zelboraf reduced the risk of death by 63%.
While many patients experience a striking reprieve while taking this drug, for most, the melanoma becomes resistant, making recurrence of the cancer likely. On average, patients in Zelboraf's clinical trials showed recurrence of tumor growth after seven months.
"The experience with Zelboraf shows that we're on the right track but that we still have to discover a way to ensure the drug remains effective over time," said Turnham. "The average advanced melanoma patient's life expectancy is still being measured in months and by testing promising treatments in combinations, we think we can extend the lives of thousands of melanoma patients."