Biotech Notebook: Onyx, Genentech, Cardiome

The stock closed Tuesday at $72.84.

Ende's analysis sounds about right to me, although I give much better odds for approval than his 20%. I see it more like 50-50, at least.

My bullishness here hinges on the assumption that the FDA considers progression-free survival -- rather than overall survival -- an approvable endpoint with real clinical benefit for these breast cancer patients. This assumption was a matter of debate at the December advisory committee meeting that voted 5-4 to recommend against Avastin's approval.

Despite that negative vote, there was plenty of support voiced by oncologists that in these breast cancer patients, a doubling of progression-free survival is a real benefit. An improvement in overall survival might be the gold standard measure of efficacy, but it's also something very difficult to achieve and demonstrate in these patients.

The Cancer Letter devoted a portion of this week's issue to the Avastin debate, quoting Indiana University's Dr. George Sledge, a professor of pathology and laboratory medicine and chairman of the breast committee of the Eastern Oncology Cooperative Group, which ran the Avastin study that forms the basis of the application in front of the FDA right now.

"This is biological reality that bevacizumab prolongs progression free survival in breast cancer when you combine it with a taxane in the front-line setting," Sledge said, according to the newsletter.

"If you believe in your heart of hearts that only drugs that improve overall survival should be approved in metastatic breast cancer, then the power of these trials for overall survival probably would require two to three times as many patients as were enrolled in these trials," he continued. "That's a very high bar. If drug companies are required to pass that bar to get a drug approved in front-line metastatic breast cancer, they will probably stop doing studies in front-line metastatic breast cancer."

One scenario I hope doesn't play out is something that my colleague Justin Ferayorni outlined in his Genentech-Avastin column on RealMoney.com last week. He highlighted the possibility that the Centers for Medicare and Medicaid Services, the government's health care reimbursement agency, may attempt to persuade the FDA to reject the breast cancer indication because Avastin's benefits in this setting (progression-free survival but no overall survival) may not outweigh the drug's high cost.

That scenario is scary, because until now, the price of a drug or a cost-benefit analysis is not something that the FDA considers when making an approval decision.

I think Justin is right that these sorts of analyses may one day play into drug approvals, but I disagree with him that the trend will start with Avastin, or with cancer drugs in general.

Cardiome in Limbo

One last word on Cardiome(CRME) and the long wait for an FDA decision on its hearth rhythm drug Kynapid: I did hear back from Cardiome President Doug Janzen last week. Unfortunately, he had little to say to advance the story.

As far as Janzen understands the situation, there have been no new requests from the FDA for new clinical data, nor have there been requests for new analyses of old data. At this point, the Kynapid review seems to be stuck in an FDA procedural logjam, he says.

While we wait, Cardiome shares continue still trade under $6, less than half of where they were three months ago.

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