They can't be trusted.
Both these answers make a lot of sense. Thank you very much. I had not thought of this.
Does the FDA want to keep its options open? If there was no definitive vote, then the FDA isn't going against the Adcom. I wonder if this was BMRNs plan from the start. Avoid head to head comparison with Eteplirsen, avoid a black and white up/down vote for approval, keep things murky.
I heard the testimony of patients who took the drug and got benefit, then stopped taking the drug and went into decline, then started taking the drug again and had ability restored. So it seems to me that it works for at least some patients.
Don't get me wrong, I think Sarepta's Eteplirsen is a far superior drug in every way, but on the off chance a patient doesn't respond to Eteplirsen, they should have a choice.
To your point, I guess you could make a good argument that if Drisapersen is approved, some will take it instead of the far safer, far more effective Eteplirsen, and that would be bad for patients. So if there was a way to approve Drisapersen but make it a treatment of last resort only, it would give people one more chance at help.
SRPT will be fine because they did the follow on confirmation study with a much higher study size - in direct response to the FDA's recommendation.
They will not get rejected because while their original sample size was small, the follow on confirmation study was consistent in the results - high efficacy, no adverse events, no side effects.
That said, I was there at the Adcom today and heard some heartfelt stories from Mom's whose sons were on Drisapersen - then off it (due to adverse side effects) then back on it again, noting that when their boy was on it, they saw the decline stop. So we have to think it works for at least some kids.
If I were in charge of the FDA I would approve both Drisapersen and Eteplirsen because patients need choices (and I'm long on SRPT, have no position in BMRN, and think Eteplirsen is a much better solution - so if I'm recommending approval of Drisapersen and I'm very biased, hopefully the FDA will approve it).
These boys need hope - they need choices!
I would love to see every one of these beautiful children get eteplirsen immediately. Their story is so gut wrenching and their smiles so beautiful and full of hope ... Words fail me.
Every single Mom who testifies brings tears to my eyes.
I am praying for each and every one of these boys and their families.
It reminds us that these are real kids and their lives are at stake. VERY powerful.
I love the smell of manipulation in the morning.
Smells like - Victory!
While I understand your point, it would be a cruel irony if it helped get Drisapersen approved, then they found some reason to not approve Eteplirsen. I wonder if the FDA indicated to her that they plan to approve both?
Looking at the M&A targets in the biopharmaceutical space, several of the big ones have announced they are looking for small/niche companies to acquire. If GSK doesn't buy SRPT, someone else will. With that in mind, GSK could easily pick up SRPT and recoup much of its investment that went into the failed Dris.
GSK would be foolish to allow Pfizer to make the acquisition, so as a blocking takeover move, it is much smarter. Meaning, cheaper to acquire SRPT than to have to fight Pfizer/SRPT.
As for BMRN, I agree with you that after walking away from Dris they aren't likely to go back and pick up the whole company at a premium, especially when they can buy SRPT and simply knock out BMRN. So your logic is sound and I agree.
Think about it, GSK could buy BMRN and SRPT with their lunch money. So they could easily wait until the Adcom /FDA approval then buy the company outright in a stock swap and then THEY would own the DMD space in its entirety.
3-months later, I'm still waiting. What a train wreck of a Beta and of a company.
Shame, the product had such potential, they just can't execute.
I agree. He came across very professional, knowledgeable, and was a good corporate representative.
Agreed. The selloff was widespread after Hillary ran her mouth, harming the entire sector. Cramer used to be a big fan of ISIS then around $70 he stopped recommending it. I'm guessing it was his Hedge Fund buddies told him they were selling off some, which became a self fulfilling prophecy. Anyway, I fully agree that nothing has fundamentally changed with ISIS or the outstanding prospects going forward. Still the best pipeline in the industry!
Let's just hope Cramer doesn't weigh in, as he is the kiss of death no matter what he says.