The Food and Drug Administration "denied the dispute appeal request," according to the company, but "to say that the FDA rejected the appeal is ludicrous." Confused? Same here. The first quote comes from the company's press releas, the second from Cell Therapeutics's CEO James Bianco during the conference call with analysts.
During the appeal process, the FDA didn't agree that the company's cancer drug pixantrone was effective, but it didn't completely shut the door on an approval, either. Instead, the Office of New Drugs said Cell Therapeutics could resubmit for approval if it can satisfy two issues.
First, Cell Therapeutics stopped its trial of pixantrone early because it couldn't enroll enough patients. The FDA applied what's called "statistical spending" to the study, deciding that the stoppage was an interim look, which made the FDA apply more exacting standards to determine whether the trial succeeded.
To understand how this works, imagine that you flip a coin 100 times. By the end of those flips, the ratio of heads to tails will likely be close enough to 50/50 to persuade you that your coin wasn't rigged to land one way or the other. But if you look at your heads and tails totals a few times while you're in the process of making those 100 flips, you might find a time when you have significantly more heads than tails. The more looks you take, the more likely you are to find a difference, so the data requires statistical spending to compensate for those additional looks.
Cell Therapeutics claims that its look was the final one, and therefore requires the regular statistical threshold. If it can convince the agency that's the case, it can get the FDA to review the data again.
Second, investors need to focus on whether the trial was sufficient to convince the FDA that the drug works well enough to warrant an accelerated approval. The standard for accelerated approvals, which only require one pivotal trial, is fairly high. After post-approval studies on Roche's Avastin and AstraZeneca's
And of course, there's the issue of having to reapply to the same agency that previously rejected your last application. Besides Gilead Sciences'
Cell Therapeutics' ball may be headed for the end zone, but the agency has an eight-foot free safety ready to bat it down.