Response from Fred Dumais re: current status ... content edited for clarity.
Original here.
___________________________________________
Subject: BLA for PG deficiency
' Hi Fred,
—Could you please clarify where we are holding at this moment wrt BLA on PG deficiency. In particular, are we still expecting the FDA to decide on priority review and/or pediatric vouchers or is that issue a definite no, since the FDA did not respond on schedule (at 60 days?)?
- Both the pediatric designation and voucher (at time of approval) are still very much at play here.
—Or are we still waiting to hear from the FDA on these two matters with the assumption that the FDA was delayed and will still get back to PLI on these with a positive response?
- We hope to soon be in a position to provide an update on the whole process.
—Do we have a PDUFA date from the FDA?
—When do we expect to get this if we do not have this yet?
- That’s the kind of info I can’t provide at the moment. Again, hopefully, we will have more soon to provide as an update
—Have we received and request for more info from the FDA that may be delaying things?
—Does PLI still think that we should see FDA approval in Q4 or has this now been delayed to Q1 2018? If so why the delay?
- I still think we have a very decent shot at getting plasminogen approved before year-end
—Do we know by when the FDA will decide on whether there will be an ADCOM for this BLA?
- That has not been discussed to my knowledge
—I am afraid that with no or very little news flow of significance (that we do not already know or without attached dollars), the shorts will continue to control. Do we know (or suspect) the purpose of these shorts?
- Fairly obvious on this one ........ They made a killing by shorting the sector (Valeant, Concordia and many others. If you win 8 times out of 10, why would you stop? In our case, the pipeline is real. Let’s get the products to market and they will go away. Are they just out to make some dollars on a company that got too fat without the revenue backup, or are they building a base and accumulating as cheaply as possible for a future attempt tp take over the co should 4050 succeed in trials? The shorts are not interested at taking over companies and I don’t believe in conspiracy theories at this time. Once we get to $200M in yearly revenues with 2-3 products approved it will be a different game but now, it’s still too early in my opinion.
Best regards '
___________________________________________
— FYI, also see — What Is a PDUFA Date?
Jim |
