Investors Need a Peephole Into FDA Offices

Cell Therapeutics (Nasdaq: CTIC) is one step closer to resubmitting its marketing application for its non-Hodgkin's lymphoma drug, pixantrone. The biotech said yesterday that it had met with the Food and Drug Administration to get guidance on what it needs to do to gain approval for pixantrone.

Boy, do I wish I could have been a fly on the wall in that room.

Cell Therapeutics went over the heads of the FDA's Division of Oncology Drug Products, or DODP, to appeal the complete response letter. The Office of New Drugs, or OND, didn't overturn the original ruling and approve the drug. But as Cell Therapeutics tells it, the OND did say that the DODP needs to look at the data again.

As I see it, the meeting between Cell Therapeutics and the DODP could have gone down in one of two ways. The OND could have come down hard on the DODP internally, and Cell Therapeutics is well on its way to gaining FDA approval. Watching the FDA admit that it's wrong would be worth the price of admission.

Alternatively, Cell Therapeutics is presenting a rosy picture of the OND's opinion, and the entire meeting was spent with the DODP's personnel rolling their eyes wondering why the heck they're sitting in a meeting discussing refilling the marketing application without a new clinical trial for pixantrone.

The problem for investors is that there's no way to know which has occurred. The FDA doesn't discuss unapproved medications, so we're only getting one interpretation of the situation.

There are two major sticking points for an approval: whether the clinical trial being used for approval showed a statistically significant benefit for pixantrone and whether it's sufficient for an accelerated approval. The likelihood of changing the DODP's mind on the first one seems highly possible.

The accelerated approval is where Cell Therapeutics will have the hardest time because it's somewhat subjective. The DODP turned down Dendreon's (Nasdaq: DNDN) Provenge the first time and refused to even look at Roche and ImmunoGen's (Nasdaq: IMGN) T-DM1 application for accelerated approval. It did give Roche's Avastin and AstraZeneca's (NYSE: AZN) Iressa accelerated approvals, but both produced post-approval results that didn't support their initial accelerated approval.

We'll know how well the meeting really went in less than a year. Cell Therapeutics plans to resubmit its application by the end of the year, which would put an FDA decision in the first half of 2012.

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