Replies to post #141865 on Biotech Values

[mcbio]

Though Pfizer performed a radiographic analysis in one tofacitinib study, the agency perspective on these data was less than sanguine however. On pages 14-18 of the FDA briefing document on the Pfizer data, there were 5 concerns raised regarding these data (see document text). Thus the agency concluded that "it is not possible to make definitive conclusions about the treatment effect of tofacitinib on structural damage progression. This is particularly important in determining the overall benefit-risk profile of tofacitinib, which is associated with serious safety concerns..."

Is it safe to assume that AZN/RIGL incorporated a radiographic study into the ongoing pivotal fostamatinib trials?

[genisi]

... the agency concluded that "it is not possible to make definitive conclusions about the treatment effect of tofacitinib on structural damage progression.

Think the FDA knows that radiographic progression is a difficult endpoint (although I agree, very important for long term efficacy evaluation in RA, but I will add that the other endpoints are no less important). While data were not definitive in the radiographic trial, the higher dose did show a stat-sig effect (although due to small subgroup), and the trend was positive in the lower dose as well, plus there was very little structural progression in the placebo group. Given the above, I think the FDA sees the overall efficacy positively and will approve in patients who failed anti-TNF therapy as they also have an unmet need.

[DewDiligence]

I think Tofacitinib has an 85% chance of approval by the Aug 2012 PDUFA date. Basically concur with genisi’s assessment in #msg-75539892—i.e. the unmet medical need will trump the ambiguity wrt Tofacitinib’s results in the (non-primary endpoint) of radiographic progression relative to the control arm.

I expect the FDA to grant a third-line label; if the FDA grants a second-line label, it will be a pretty big win for PFE, IMHO.