Can anyone explain to me why the FDA would grant orphan drug approval when this give 7 year exclusivity to Sarepta? Wouldn't this block other companies with potentially better treatments from entering? I think based on this the FDA will hand out a CRL.
What does going to the sideline mean? They should just admit it and downgrade to an actual price...but I suspect the company is working on a raise with Oppenheimer as we speak.
No it's folly to think the FDA goes against staff and the panel. Those are rare instances...so you are betting on a an less than 5% chance of approval (instances/ all adcoms). It's not looking good.
Anyone can still short this down to cash value and some more. Sarepta will need to raise cash before PDUFA to continue their phase 3 which they will need to amend protocols guaranteed. Also, watch for the CEO/CMO to be let go before this happens.
The phase III needs to have its protocol amended and dosing increased asap! They also need to change how they test for dystrophin and the location of the biopsies...etc. Lots of problems that need $$$ to fix.
And how would she approve it? To congress, I have made my decisions as the director of CDER to approve ....while my staff have found no signs of efficacy that meets the requirements of substantial evidence as well while an independent panel has voted against AA and full approval, I believe this approval will make you and I look good. Of note, they have orphan status which means they can sell this ineffective drug for 7 years exclusively at 300K/patient???? Get real man. Stop pumping and stop deceiving other investors.
You have to talk about the clinical endpoint first. If they don't meet it then you talk about totality of studies to day with secondary endpoints. If that fails then no AA as well no approval. You don't understand biotech.
2 in XXX? It's a low percentage bet...not worth it. But according to Needham and Oppenheimer TP is still more than $40. Idiots...
To hear this from several deputy directors and repeatedly means Sarepta mislead investors. Sarepta flexed their lobbying muscles and tried to bully the FDA with politicians who i don't believe were informed either. It's unethical of Sarepta because now think of how many patients will suffer.
The data wasn't good at all. All decisions are to be made on available clinical data so it was down to clinical data vs. anecdotal. The panel and staff went with clinical instead of the 51 anecdotal testimonies.
No...just stop. Did you watch the adcom? Panel members and the FDA staff could not interpret the data. Woodcock won't overturn both her staff and the panel.
Add Needham to the list of idiot analysts you should NEVER listen to...
Correction, big pharma was watching until they saw how bad the data was.....how do you value a company with data that doesn't prove the drug works? You can't, so stop deceiving people.
This is what's going to happen, usually does for all biotech companies. Sarepta will PR and try to lay blame on the FDA and not on their inability to run a proper trial. The media will run neutral stories to protect the FDA and provide some encouragement to the families affected. Analysts will downgrade (Marai may raise PT again cause he's an idiot) to cash value or discounted cash. Look for the really smart analysts who downgraded just after the BD release they'll remain truthful while Oppenheimer will try to fix their reputation with some BS. It won't recover leading up to the PDUFA date because the stock is reliant on this one drug without anything in the pipeline that will offer a good return in the short term. Sarepta may try to raise cash before PDUFA because it's their last chance before prices plummet. Sarepta may let their CMO/CEO go because of this failure and as a condition to receive more money. I suspect Woodcock wiill reject this because the staff and the panel doesn't support efficacy. I would have concluded differently had Bastings, Temple, Dunn not rambled on about substantial evidence and stories of how the FDA saw historically controlled trials fail. I also noticed Bastings was extremely frustrated and upset at Sarepta's misleading statements. Farkas also was very accomodating and the FDA disclosed that they had tried to help Sarepta by blinding their reanalysis (hello? incompetency on SRPT to have the FDA help you on). Institutional buyers will dump their positions before PDUFA to the hopeful. My advice, don't be hopeful.
MR MARAI!!!!!!! Is it a fire sale yet or are you raising price to 90? Seriously you know how much money people will lose because of you?
Nope...she is there for crowd control. Her answer to Congress now is that staff did not find signals of efficacy and panel voted no.
People like you are uninformed and invite more shorting. Game over. You can buy and wait for the phase 3 but that needs to be amended to meet the fda requirements.
No...you should have watched the adcom. Dr. Dunn, Temple, Farkas and Basting ripped sareptas small data set apart. No chance of an approval...you'll only lose more money. Trust me on this woodcock was there to keep the order only. Her answer to Congress will be...panel voted no and my reviewers found no signal of efficacy. Saepta misled the agency. Done closed. End of story.