Melanoma has been considered one of the toughest cancers to treat, with few drug options…until now. The FDA has approved Roche’s unique acting drug for melanoma, the deadliest form of skin cancer. Zelboraf (vemurafenib) works by targeting a mutant gene that’s found in about half of melanoma patients. This makes two drugs approved that direct confront the deadly disease, a form of skin cancer.

Zelboraf will be available within two weeks according to Roche.

Dr. Anna Pavlick, director of the melanoma program at the New York University Cancer Institute and one of the scientists involved in research on the drug stated:

“Zelboraf is a huge step forward in the fight against metastatic melanoma, and the advancement of treatment for the deadliest form of skin cancer.”

Melanoma is the fastest-growing form of cancer in terms of new diagnoses. Scientists attribute the acceleration to longer life expectancies in the U.S. and increased indoor tanning by the young. About 68,000 people in the U.S. were diagnosed with melanoma last year and 8,700 died, according to the American Cancer Society.

Zelboraf will provide a second option for melanoma patients with a mutated form of a protein called BRAF that helps with cell growth when working normally. Zelboraf slows tumor growth by blocking the mutated form of the protein.

Roche estimates that about half of all melanoma patients have the BRAF mutation. Mutations in the BRAF gene can cause disease in two ways. First, mutations can be inherited and cause birth defects. Second, mutations can appear later in life and cause cancer, as an oncogene.

Hal Barron, M.D., chief medical officer and head of Roche’s Global Product Development comments:

“The FDA approval of Zelboraf marks a major step forward in personalizing the treatment of metastatic melanoma, a devastating disease that until this year had limited approved treatment options. We will continue to study this medicine with a goal of further improving outcomes for people with melanoma and other cancers that are driven by BRAF mutations.”

Inherited mutations in this gene cause cardiofaciocutaneous syndrome, a disease characterized by heart defects, mental retardation and a distinctive facial appearance. Acquired mutations in this gene have also been found in cancers, including non-Hodgkin lymphoma, colorectal cancer, malignant melanoma, papillary thyroid carcinoma, non-small cell lung carcinoma, and adenocarcinoma of lung.

In March 2011 the FDA approved a Bristol-Myers Squibb drug, Yervoy, which was the first drug shown to prolong survival in patients with advanced skin cancer.

The FDA approved the drug on the basis of a 675-patient study in which patients received either Zelboraf or an older chemotherapy drug. Among people in the ongoing study, 77% on Zelboraf are alive versus 64% of those taking the older drug, the FDA said.

Despite the higher survival rate, melanoma adapts quickly, and patients saw their tumors resume growth after seven months, on average.

Side effects with the drug included skin rashes, joint pain, fatigue, diarrhea and hair loss. About 26% of patients developed a less serious form of skin cancer.

Timothy Turnham, director of the Melanoma Research Foundation is elated by the approval:

“The FDA’s quick action on this drug approval is important because it gives melanoma patients a new way to fight this deadly disease.”

The approval comes months before the previously estimated date of October 28, 2011.

Written by Sy Kraft