FDA's decision to delay a ruling on whether it should pull the metastatic breast cancer indication from the label for Roche/Genentech's Avastin is stirring hope that there could be a positive outcome - though for an agency wrestling with a controversial issue, it is the only prudent decision.

Regulatory action on a supplemental BLA to convert the accelerated approval of Avastin (bevacizumab) for previously treated advanced HER-2 negative breast cancer into a full approval had been due on Sept. 17, but FDA says it needs more time, Genentech said in a late-afternoon statement.

The delay could be a good sign for Genentech in that it shows the regulator is taking time to carefully consider the decision and information submitted as part of a recent review of the BLA ("Avastin Approval Debate Pits Clinical Benefit Versus Statistics," Also see "Avastin Breast Cancer Approval Debate Pits Clinical Benefit Versus Statistics" - Pink Sheet, 26 July, 2010.).

In its statement, Genentech said it was "pleased by the FDA's decision to review additional information on Avastin," and that it will work closely with the agency during the review period.

Roche's largest drug, Avastin is approved for many indications, including colorectal cancer, non-small cell lung cancer and renal cell carcinoma.

Avastin had been granted accelerated approval for the breast cancer indication in 2008. But in July, FDA's Oncology Drugs Advisory Committee voted unanimously against full approval after data were released showing a smaller benefit for progression-free survival, no benefit for overall survival and troublesome side effects.

The advisory committee vote on the breast cancer indication has proven to be very controversial and has generated enormous media coverage. Breast cancer patients who dramatically benefited from the drug have petitioned FDA to keep the indication and have been featured in local and national media (Also see "Avastin Reimbursement Worry Drives Patient Petition To FDA" - Pink Sheet, 2 August, 2010.). Sen. David Vitter, R-La., jumped on the issue to warn of early signs of rationing as part of President Obama's health reform.

Christi Turnage, a breast cancer patient who launched an online petition against withdrawal that has over 7,000 signatures, said she is hopeful that the FDA is not rushing into a decision and instead carefully considering the additional information provided by Roche/Genentech as well as the effect of a withdrawal on patients. A stack of testimonials from patients who signed the petition shows that some women are benefiting enormously - the trials may not prove that, but "that's no reason to kill the drug," she said in an interview after the delay was announced.

However, given the unanimous negative vote by ODAC, Avastin's prospects in breast cancer don't look very rosy. Experts expect FDA will likely follow ODAC's recommendation and remove the metastatic breast cancer indication from Avastin's label, observed Bernstein analyst Timothy Anderson in a Sept. 2 note. This will lead to less favorable positioning on compendia by the National Comprehensive Cancer network and other groups, he pointed out. A withdrawal could also affect the future of Avastin in development for other indications, such as ovarian cancer, he continued, and raises questions about the use of PFS as a surrogate endpoint in trials.

In addition to a complete withdrawal of the indication, FDA also has the option of making an exception for patients who are already taking and benefiting from the drug.

- Emily Hayes (e.hayes@elsevier.com )

Roche Gets Reprieve On Avastin For Breast Cancer With Delay At FDA

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