FDA approval of Roche melanoma drug may come early

LOS ANGELES Wed Aug 10, 2011 1:53pm EDT

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LOS ANGELES (Reuters) - U.S. regulators are moving quickly with Roche's application for targeted melanoma drug vemurafenib, which could receive approval as early as this week, according to a source familiar with the situation.

A pivotal trial found that advanced melanoma patients taking the experimental pill, being developed in partnership with Daiichi Sankyo, were 63 percent less likely to die from the disease than patients given chemotherapy.

Vemurafenib, whose brand name is Zelboraf, is designed for patients with tumors that have a mutation in a gene known as BRAF that allows melanoma cells to grow. About half of all melanomas, the deadliest form of skin cancer, have the genetic aberration.

Roche filed for U.S. approval of the drug in April and the Food and Drug Administration is slated to decide on the application no later than October 28. Approval of a test to detect the BRAF mutation is expected at the same time.

"We are getting the sense that approval is imminent," said the source familiar with the matter.

An FDA spokeswoman declined to comment on the timing of a decision, but did say the deadline is "a performance goal post" for the agency, which in the past has approved medicines ahead of time.

In late April, the FDA approved Johnson and Johnson's prostate cancer drug Zytiga about two months before the scheduled deadline.

In March, the FDA approved the first treatment to help patients with advanced melanoma live longer. The drug Yervoy, or ipilimumab, is sold by Bristol-Myers Squibb.

Yervoy, which posted better-than-expected sales in the second quarter and costs about $120,000 for a course of therapy, is an antibody designed to spur the immune system to fight off the melanoma.

Analysts have forecast annual Zelboraf sales for Roche of $732 million by 2015, while Bristol's Yervoy sales are expected to reach $1.43 billion, according to Thomson Reuters data.

Roche and Bristol have also agreed to collaborate on a novel clinical trial to see if the two melanoma drugs are safe and effective if taken together, potentially allowing them to be prescribed as a drug cocktail.

COMBINATION THERAPY

Roche's Genentech unit, which will market the cancer drug in the United States, declined to comment on price. The company estimates that around 9,500 of the 70,000 new cases of melanoma expected to be diagnosed in the United States this year will be advanced forms of the skin cancer, with the BRAF mutation found in about half of all melanoma cases.

The diagnostic test will cost around $150, according to Paul Brown, president of Roche Diagnostics.

"This is really now the exemplar of personalized healthcare ... science and our understanding of disease has fundamentally changed and grown over the last 5 to 10 years," he said.

If approved, the medicine would be the first new oncology product from Genentech since 2004's launch of lung cancer drug Tarceva.

Dr. Jeffrey Sosman, director of the melanoma program at Vanderbilt University in Nashville, Tennessee and a vemurafenib trial investigator, said the drug "will have an immediate impact on many patients with melanoma," particularly those with aggressive disease.

"Ipilimumab has very different characteristics, and in patients with disease that is not so aggressive and you have a much longer period in which to follow them, ipi is a good alternative and could be given initially," he said.

Sosman also said patients eventually become resistant to Zelboraf, and there is a need for more investigation of combination therapies.

"I'm alive," said 52-year-old melanoma patient Susan Steel. "I probably wouldn't be without this drug."

Diagnosed with melanoma in 2005, Steel has been on vemurafenib since January under an expanded access program and seen her tumor volume shrink as much as 80 percent.

Side effects have included rash, slight hair loss, extreme photosensitivity and joint pain.

"All of this is irrelevant," Steel said. "I can still talk. I can still make speeches, take care of my children ... I can listen to all the reasons why my daughter doesn't want to complete her college applications. That was a gift."

(This story corrects the filing timeline in paragraph 4 from May to April)

(Reporting by Deena Beasley; Editing by Michele Gershberg and Gerald E. McCormick)

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