Roche wins early approval for blockbuster melanoma drug vemurafenib

In a rare move, the FDA has stamped an early approval on Roche's remarkable new melanoma drug, vemurafenib, giving it a green light on a potential new blockbuster two months ahead of its PDUFA date. For Roche and Daiichi Sankyo, which will market the drug as Zelboraf, the approval marks another key milestone in the advancement of new cancer drugs specifically targeted to the molecular triggers of the disease.

"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," said Dr. Hal Barron, chief medical officer at Roche. Roche plans to sell a 6-month course of treatment, which targets the mutated BRAF gene, for $56,400.

The drug was originally developed by Plexxikon, a Berkeley, CA-based biotech we selected for the Fierce 15 in 2010. Faced with a chilly public market for biotechs Plexxikon opted to be bought out by Daiichi Sankyo in a $935 million deal, pushing ahead as a wholly-owned cancer drug development subsidiary. Roche snagged its marketing rights in a collaboration pact inked back in 2006.

In clinical studies researchers reported that the drug reduced the risk of death in its targeted population by 64%, a stellar result for the cancer field. Anna Pavlick, director of the melanoma program at the New York University Cancer Institute, told the Wall Street Journal that the data "takes your breath away if you're a melanoma doc."

The early approval was first flagged by Reuters several days ago.

"This has been an important year for patients with late-stage melanoma. Zelboraf is the second new cancer drug approved that demonstrates an improvement in overall survival," said Dr. Richard Pazdur, director of the Office of Oncology Drug Products in the FDA's Center for Drug Evaluation and Research. "In March, we approved Yervoy (ipilimumab), another new treatment for late-stage melanoma that also showed patients live longer after receiving the drug."

- here's the FDA release
- read the WSJ story