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New Zelboraf data shows big survival boost

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Roche's ($RHHBY) new melanoma drug Zelboraf apparently works even better than researchers suspected. Approved by the FDA via its fast-track program, the targeted therapy demonstrated early on that it improved survival time for patients with BRAF-positive melanoma. However, newly published trial data highlight median overall survival of almost 16 months, compared with 6 to 10 months for the typical patient with metastatic disease.

"This study shows that Zelboraf changes the natural history of this disease," study co-author Dr. Antoni Ribas said, as quoted by the BBC. "This data is beyond what I would have expected. We're seeing a significant number of patients with durable responses to the drug, and that the whole group of treated patients is living longer."

But questions do still remain. The trial included only 132 patients, all with the BRAF mutation, and all of them were given Zelboraf. But it confirmed response rates found in Phase I research: The overall response rate was 53%, with 6% enjoying complete response and 47% partial response. Patients gained 7 months of life, on average, a big step forward for melanoma, explained lead author Dr. Jeffrey Sosman. "This is the first time we have shown such a long extension in survival," Sosman said.

The hope is that Zelboraf will be even more effective when given to patients earlier in their disease course. This trial included patients, mostly men, with Stage 4 disease. "If we use it in an earlier stage, then we are hoping to cure patients who would not be cured otherwise," Sosman said.

Cost could be a deterrent, however. Sosman figured patients would need to use Zelboraf "for an indefinite period of time," and at $10,000 a month in the U.S., that's a hefty bill, even if it is comparable to other survival-enhancing cancer drugs. In England, health officials are apparently anticipating some sort of discount. "It will be interesting to see what price the manufacturer charges so as not to place too great a strain on already scarce NHS resources," Elizabeth Woolf of Cancer Research UK said, as quoted by the BBC. The drug was just approved in Europe earlier this week.

Special Report: FDA approvals of 2011

- get The New England Journal of Medicine abstract
- see the HealthDay story
- get more from Medscape
- read the BBC coverage

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