We haven't seen the early-stage data yet -- it'll be presented at ASCO in June -- but the trial was fairly straightforward. Patients either got ipilimumab and dacarbazine, or dacarbazine alone. The combo treatment extended survival by more than the chemotherapy alone.
The trial that supports the application for treating late-stage melanoma is a little more complicated. Bristol combined ipilimumab with an unapproved peptide vaccine called gp100, then compared the combo treatment to each single treatment. Ipilimumab by itself, and in combination with gp100, performed much better than gp100 alone. But without a true placebo control, the argument can always be made that the difference in survival was because gp100 had a negative effect on survival rather than a positive effect by ipilimumab.
The FDA didn't hold an advisory panel to review the data with a panel of outside experts, so the agency is clearly confident in its decision. The question is, in which direction? Is it perfectly fine with the gp100 control, or does it think the data is clear enough that it doesn't need experts to tell it that?
I have to think it's the former. The data is solid enough that I have a hard time seeing the agency rejecting it outright without at least listening to outside experts. And besides, even in cases like Cell Therapeutics'
Of course, something minor could always trip up ipilimumab and help drugs such as Vical's
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