Roche and Exelixis roll toward FDA approval with a melanoma cocktail

Roche ($RHHBY) and Exelixis ($EXEL) are one step closer to FDA approval for their skin cancer-treating combination treatment, as the agency has accepted their application and promised a speedy review.

The companies are seeking approval for a combination of the Exelixis-discovered cobimetinib and Roche's on-the-market Zelboraf, a cocktail that targets melanoma patients with the V600 mutation in their BRAF genes. Roche's Genentech unit licensed cobimetinib back in 2006, and, if the combo wins approval, Exelixis is entitled to a 50% share of U.S. sales plus royalties on global revenue.

The agency has granted the combination its priority review status, cutting four months off of its standard timeline and promising to hand in a final decision by Aug. 11. Roche and Exelixis are also awaiting European approval after filing an application in September.

Each filing is based on Phase III data in which the combination helped subjects live significantly longer without tumor growth than those treated with Zelboraf alone, hitting the primary goal of progression-free survival (PFS). Patients treated with the combination charted a median PFS of 9.9 months compared to 6.2 months on Zelboraf, the companies said.

Exelixis CEO Michael Morrissey

"The FDA's acceptance of the cobimetinib NDA brings us one step closer to a potential new treatment option for patients with advanced BRAF V600 mutation-positive melanoma, a form of the disease for which new approaches are needed," Exelixis CEO Michael Morrissey said in a statement.

The news sent Exelixis' shares up nearly 10% in premarket trading on Thursday, but pervasive setbacks with the biotech's wholly owned pipeline have marred investor confidence. The company's lead asset, cabozantinib, failed to beat the immunosuppressant prednisone in a Phase III trial in prostate cancer last year, results that sent the biotech's shares plummeting. Exelixis promptly moved to cut about 70% of its workforce, paring down its operation while holding out hope for two ongoing studies testing cabozantinib's effect on kidney and liver cancers.

Exelixis came up with cobimetinib and advanced it to the IND stage before signing on with Genentech, reaping $40 million up front. The drug works by shutting down the kinase MEK, a pathway that regulates growth factor receptors and plays a hand in helping tumors proliferate.

- read the announcement

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