Biotech Values

[DewDiligence]

PTLA—The PR headline says this trial is under the Accelerated Approval pathway, but I wouldn’t interpret that as a binding commitment by the FDA to grant AA if the study results are positive.

As an addendum to the above (#msg-99095723), I was thinking about what drugs the FDA has approved based on a single phase-3 trial in healthy volunteers with N<=64.

I couldn’t think of any, so it looks like PTLA’s phase-3 trial is sui generis.

The design of the andexanet alfa phase-3b/4 outcomes study in oral-FXa users who encounter a major bleeding episode has not yet been established and would seem to be a Herculean task. However, if PTLA’s Accelerated Approval strategy works, andexanet alfa will be approved long before the results of the outcomes study will be reported, and this is one case where the FDA might be reluctant to pull an AA drug from the market.

The entire scheme sounds a little too convenient for PTLA, which is why I’m somewhat skeptical.

p.s. PTLA’s ASH webcast from 12/9/13 (~60 minutes) is well worth a listen for anyone who has a position or is thinking of establishing one: https://event.webcasts.com/viewer/event.jsp?ei=1025469 .