PCYC... I file on you.
Pharmacyclics to file cancer drug over FDA protest
Wed Apr 4, 2007 8:00am ET162
Reuters By Bill Berkrot
NEW YORK, April 4 (Reuters) - Pharmacyclics Inc. (PCYC.O: Quote, Profile , Research) has decided it will not take no for an answer.
The small biotechnology company said on Wednesday it was taking the unusual step of filing its application for an experimental cancer treatment with the U.S. Food and Drug Administration "over protest."
"I want them to accept our NDA (new drug application) and look at the data carefully. I think that the data supports safety and efficacy and I think it should be approved," Pharmacyclics CEO Richard Miller said in a interview.
The FDA in February rejected Pharmacyclics' application to review the drug, Xcytrin, for the treatment of lung cancer that has spread to the brain -- news that nearly halved the company's share price.
In its "Refuse to File" letter, the FDA said the company's application was not complete enough to permit a review based on the clinical studies in which the drug missed the primary goal that measured the progression of neurological symptoms.
Filing over protest is a procedural move permitted by FDA regulations that allows a company to have its NDA filed and reviewed when there is a disagreement with regulators over the acceptability of the NDA submission.
"This is not the way you want to do things, but we feel that we've been treated unusually here," said Miller, who said the company was "very surprised" by the refuse to file letter.
Miller, who is still a practicing oncologist, feels the drug got short shrift from the FDA, noting that the refuse to file is usually reserved for problems with the application itself and not about whether a drug met its primary end point.
"The oncology division at the FDA has been progressively getting more and more strict over the last three to five years," Miller said.
"The pendulum has swung to the point now where they're saying if you don't have (statistical significance) on the prespecified primary end point, we're not even going to look at it," Miller said. "All we are asking for is that people take a look at the totality of the data very carefully, not just one number."
There is precedence for drugs that were filed under protest eventually receiving approval. Miller noted GlaxoSmithKline's non-Hodgkin's lymphoma drug, Bexxar, as such an example.
The Xcytrin filing date will be on or about April 30, which is 60 days after the company's request for a meeting with the FDA to discuss the refuse to file letter, Pharmacyclics said.
While there are no guarantees, the company hopes it has made a strong enough case to get an expert panel to review the Xcytrin data. Such a panel could make a recommendation for approval that would carry considerable weight with the FDA.
Miller said the late-stage studies came very close to meeting the primary goal for treatment of brain metastases. In a subset of patients treated in the United States, he said the drug was highly statistically significant in time to neurological progression -- 24 months in the Xcytrin plus radiation group versus 8 months for radiation alone.
What the FDA saw was a primary end point result that suggested no definitive proof the drug worked.
"We know why we missed our end point," Miller said. "Because a few centers in France, rather than radiating patients immediately or within days, waited weeks or months before and that skewed (the results) horribly.
"There's no question here that we have an active drug. When you look at the total package, I think this has a very good chance for approval and so do my colleagues in the medical community." |
