FDA Approves Skin Cancer Drug Zelboraf

August 17, 2011— -- The U.S. Food and Drug Administration today approved the second late-stage melanoma drug this year, adding the drug to the treatment arsenal for patients with one of the deadliest forms of skin cancer.

The drug, known as Zelboraf, could offer hope for patients with metastatic melanoma that cannot be removed with surgery, experts said.

Zelboraf, which was reviewed under the FDA's priority review program, received advanced approval after a single international study published in June 2011 showed that patients who took drug lived longer.

"Approval of this drug is extremely significant because the treatments we have had up until this year were well known to have no effect on overall survival," said Dr. Kelly McMasters, chairman of the department of surgery at the University of Louisville Hospital. "It is gratifying to see a well-tolerated new targeted therapy that is effective."

The study, funded by the drug's pharmaceutical developer, Hoffman-La Roche, followed 675 patients randomized to receive either Zelboraf or a standard chemotherapy drug. Seventy-seven percent of those who received Zelboraf were still living after eight months, compared to only 64 percent of those who received the standard chemotherapy drug.

The drug specifically targets tumors with the genetic mutation known as BRAF V600E. The agency also approved a diagnostic test that will determine whether someone has the mutation.

"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," Alberto Gutierrez, director of the FDA's Office of In Vitro Diagnostic Device Evaluation and Safety, said in a statement.

BRAF protein mutation is found in nearly half of all patients with late-stage melanoma.

"We now have the capability to analyze a patient's melanoma tumor for the genetic mutation BRAF and use the targeted treatment Zelboraf to attack the tumor, shrink it and stop the progression of this deadly disease," said Dr. Anna Pavlick, director of the NYU Melanoma Program at the NYU Cancer Institute, who has been involved in clinical trials for Zelboraf.