BMRN's CEO was completely full of it.
I knew the briefing docs would be bad, but I didn't realize they would be as bad as they are. There is no benefit-risk to taking drisapersen and no question about it at the end of the panel. The odds that drisa gets approved are next to nil. CRL with requirement to complete a new Phase 3, which BMRN has no intention of doing.
I won't feel bad if BMRN ends up losing all of its money on buying RNA. If drisa is shot down and etep approved, I wouldn't give in to royalties if I were SRPT. I'd fight it in court.
I think this will be the key to whether or not eteplirsen is approved, and a question regarding this will be what I first look for in the briefing documents. If that's there, I see no way eteplirsen isn't approved. Given the safety, I don't think there's any way a reasonable expert could say the benefit doesn't outweigh the risk.
If this question isn't present, it changes things and makes an approval a much bigger fight at Adcom. But I think the odds are that it will be present and eteplirsen will thusly be approved.
Gotta admit that I thought drisa's chances were decent, but at this point, I think it's highly unlikely it gets approved. The FDA telegraphed that with their final questions for the review panel.
It is highly unusual for the FDA to approve a new class of drug without an Adcom. I'm going with the odds on this one.
You're right. The FDA is going to suck on etep's #$%$. No way would they say anything negative about it. That would hurt invetors' wallets too much. Let's completely ignore history, once again.
Suggesting that SRPT isn't going to have an Adcom is ridiculous. Eteplirsen has had way too much controversy around it--much of it created by the FDA--that they aren't going to do an about face and suddenly skip the Adcom. What an absurd idea.
I'll take a stab.
1. N=12 Phase 2 with two boys removed from statistical analysis (i.e., they will question efficacy)
2. Percent of dystrophin produced being significant enough to warrant it as a biomarker
I think SRPT's odds of overcoming these issues are better than average, and its safety will be the shining star, but the FDA has repeatedly raised concerns about these two issues. They aren't going to suddenly forget them when the briefing docs roll around. The devil's advocate reviewer is likely to slam these two issues. I don't know why anyone would assume the FDA won't given the history here.
If eteplirsen has the same review questions as drisa at Adcom should be a concern. The FDA did not ask the Adcom whether or not they recommend approval for drisa. They did that intentionally. The key to etep's success will be foreshadowed in the question section of the briefing docs.
How does recognizing that the FDA is likely going to diss on etep mean someone is short? Most briefing docs cast heavy doubt into drugs, particularly those as controversial as eteplirsen. Be objective. Simp is probably right that briefing docs will not be pretty.
The vouchers are independent of one another. Neither drug being approved affects the voucher of the other drug. As long as etep is approved, SRPT will still receive the voucher, even if drisa is approved.
Highly unlikely. Even major pharmas can't normally launch in one month, particularly with the complicated manufacturing of eteplirsen.
Yes it does. It's not going to happen and isn't suddenly more likely to because of today. Please be realistic and objective.
Today's news is good for SRPT, but no huge surprise. Everybody expected drisa to get knocked. It's the reason BMRN hasn't dropped all that much. It was baked in. SRPT has the advantage of having a high short float, so it produced a nice pop.
The real test comes on Tuesday. I do believe the line of voting questions for the panel indicate the FDA is very weary of approving drisa. That being said, the briefing docs for eterplirsen are going to be just as harsh. It's the nature of the game. But I like etep's chances much better. I can see drisa being denied or delayed. I don't think that happens for etep, even if it is slammed in briefing docs.
Per Adam F on twitter: Muscular Dystrophy Association pitching reporters with offers to interview DMD moms of drisa patients heading to DC for $BMRN FDA panel.
Guess we'll find out what the parents of boys who had been on drisa think. I doubt MDA would be doing this if the parents were going to speak negatively about drisa.
Counterarguments are made at the Adcom based on FDA's concerns. The briefing docs should be available to the public on the Friday before the Adcom.