Back in the early spring, Cell Therapeutics sought approval from the FDA of a New Drug Application for its lead candidate, the non-Hodgkins drug pixantrone. The FDA (as well as the EMA) had already endowed it with orphan drug status. All CTI had to do was present the findings from its Phase III EXTEND trial of pixantrone, the design of which CTI and the FDA had long, long ago agreed upon.
In early spring they presented their findings to an FDA advisory panel, and the panel voted unanimously 9-0 to recommend that the FDA reject pixantrone, which it did. I wrote about the panel's response, which expressed shock and dismay over numerous aspects of the trial, including its poor design (which did not conform to the agreement) and the wildly poor performance of the drug itself. The FDA said, in sum, that CTI had to run another much larger trial of the drug and prove both safety and efficacy.
Predictably, CTI's Wall Street value plummeted. By the summer they seemed to be rolling over, and it looked like maybe a drug/biotech company might actually heed the FDA and accept that the product they developed was in truth lousy.
Well by September it was clear that the company's shareholders had made their feelings known, because CTI completely changed their tune, and formally appealed the FDA's decision, saying they decided to do so "after discussions with a number of leading U.S. and international lymphoma experts and leading biostatisticians, all of whom have reviewed the (pixantrone) protocol, statistical plan and trial results."
I looked up one of those 'international lymphoma experts' and found that while she frequently publishes in respected, peer-reviewed journals, not a single one of her papers was free of a conflict of interest, which just so happened to be sponsorships from the companies that made the drugs about which she was writing such glowing reviews.
I am neither an international lymphoma expert or a leading biostatistician. I do not have access to the raw data from the EXTEND trial (and it wouldn’t matter if I did). I can only go off what CTI presented to the panel, and what the panel then published as an FDA briefing document (opens as PDF).
In a nutshell, this abhorent company wants the regulatory agencies in the US and Europe to permit them to begin selling this drug to patients with relapsed, refractory NHL who have failed two previous therapies-- in other words, an extremely desperate patient population --- even though the best they can say about the drug is that they only know of eight people in the world who were given the drug and when tested later, showed no signs of their cancer.
Eight people ... (to be continued in Part II)
