Simply due to the snowstorm. They have no sense of urgency to do anything at all about Eteplirsen. Just another day at the office for them. Horrible isn't it?
The FDA has already denied Eteplirsen. It blows me away that some on this board are still pretending that there will be some intervention, or that the FDA will approve without Adcom, or that Congress will step in. It won't happen. The FDA is absolutely corrupt beyond all hope. It is the government spun out of control in the very worst way. While I wish it were different, this is the situation, period.
You are dreaming. The USA is the cash cow and the FDA is the gatekeeper to that. Patients aren't crossing the border to Canada for anything right now, and they won't in the future.
You made it personal when you insulted me and acted like a jerk, taking delight in my financial hardship and losses. I assure you, you would not meet me, nor would it be a nice cup of coffee. Come to the Adcom for SRPT, at least then you will be fairly safe with lots of people around.
Come meet me face to face ...
We both know you won't, which makes you a coward.
It boils my blood that you are enjoying such severe pain on my part.
I'd like to return the favor, so come meet me face to face and prove you aren't a coward.
Same argument that the FDA is using to dismantle SRPT. That the results come from critical care, not the drug. SRPTs Eteplirsen has much higher efficacy than Ataluren, yet the FDA is slamming Eteplirsen and won't approve it. Same will happen to Ataluren.
Wrong. When I invested in PTCT the P3 trials were not yet released. Further, only a complete fool would pretend that the drug didn't work, because it DID work for most of the patients. I guess by your logic, any school grade you got below 100% was a "failure". Any politician that gets less than 100% of the vote "fails".
Quit being such a jerk. Face to face you would be completely different, and that makes you a coward since you'll only rant here behind the anonymity of the Internet.
Why be such a jerk? Face to face I assure you that you would be much more polite. Silent even.
I didn't invest in a company with a failed phase 3 trial. I invested in a company with an ongoing phase 3 trial that was working remarkably well in the patients I heard about. Sadly it didn't work for every single patient in the study, as some were beyond help. - which is the only reason anybody can pretend the phase 3 trial failed - while ignoring the 85% patient population that it worked well for.
This has nothing to do with the company or the drug, it's all about the corrupt FDA.
The reason I can't short this is because the risk/reward ratio simply isn't there. If Ataluren gets approved (unlikely as it is) this stock rockets to more than $100/share, so would quadruple. If not approved, it gets cut in half so put 50% potential profit vs. a potential loss of 4x and the math doesn't work. If you can do it, great, especially since the FDA seems to be heading your way. I cannot. I've lost too much already.
While I do run a company (CEO), it isn't a bio-pharmaceutical company. If I did and I had a DMD candidate drug, I'd pull the plug on it immediately and save the money. The FDA has made it very clear that they will not approve a DMD drug no matter what. The consternation surrounding BMRN and SRPT illustrates the FDA's bias against these companies. I really thought that BMRN had a shot, even though their drug was the lesser drug, because their marketing power and influence would help. I was wrong. The FDA will not pass a DMD drug no matter what.
They said that clinical trials data was OK, but not as strong as Sarepta's results. The FDA just crushed Sarepta excusing away all of the benefit as part of critical care, and completely dismissing the drug benefit. There is no reason to believe that PTCT will not encounter the same resistance/prejudice at the FDA. Looks dismal at best. While I lost a ton of money on this stock, I feel worse for the kids doomed to die now because the FDA won't even give them a chance. It is sickening how corrupt the FDA is.
I wish you were right, but don't see it happening. SRPT will get thoroughly crushed then PTCT will. I'm still not willing to short the stock because I believe in the company, so I"ll stay on the sideline. This is a horrible situation that shows how corrupt the FDA is.
While I have full faith in PTCT, I have absolutely NO faith that the FDA can do anything right. They are absolutely corrupt and nothing can overcome that. Ataluren will not get approved because the FDA is so blatantly corrupt and PTCT will fall to the single digits.
Very sad. Get out while you can.
I didn't see that note, but given the problems that SRPT is facing with the FDA I can't see it being any better for PTCT. Yes higher number of patients, but a far lower efficacy level and if the FDA just discounts that as part of critical care, then I don't see any hope for PTCT at all. They will focus on the failed P3 trial and it will be vicious.
Based on SRPT and BRMNs Adcom, there is a 95% chance the FDA will REJECT Translarna/Ataluren.
Remember the comedian's line: You can't fix stupid. I agree with you completely biotech-expert123, SRPT efficacy was far beyond that of any other. If they are going to disapprove Eteplirsen, then Translarna has no chance at all.
I really believed they would approve both of them. For that matter, given that there are NO treatments available for DMD, I fully expected the FDA to approve BMRNs too (though I think that would have been wrong). It is unthinkable they are going to disapprove all 3.
Nobody should ever bet on the FDA doing the right thing. SRPT has much higher efficacy than Translarna did, though the patient population for SRPTs study was too small. Conversely, BMRN had a much larger study, but Drisapersen wasn't effective nor was it safe. The FDA seems to have a problem with each and every one of the drugs. Based on what we have seen so far, I see NONE of them getting approved. Not BMRN (well, they already go their CRL so that is over), not SRPT, and not PTCT.
Wrong. The best you can do is claim the small number of patients didn't show enough efficacy. It was 100% effective in the 12 patients that it was given to, and the follow on study has more than 100 patients and so far those are all both safe and effective. While small number of patients, a very long study that showed remarkable results.
As for the FDA they are absolutely corrupt, putting their own careers ahead of the needs of patients.