I'm still guessing that the FDA, upon reviewing BMRN's NDA, knew that this drug didn't stand a chance of being approved. Was the "tentative" scheduled panel review date they gave SRPT, on Jan 22nd, a tell? The FDA knows that parent advocates would be deeply disappointed if BMRN's panel was largely negative, and presaged a CRL, leaving their only hope then on just one potentially approvable drug. Will the FDA move the panel meeting date up by a few weeks, since the delay of two years has been caused in large part by FDA's glacial pace and additional hurdles placed in Sarepta's path?
The FDA owes boys with DMD, and their parents, the most rapid timeline possible to move Etep out of the clinic, and to make it available to all exon 51 amenable boys.
You're thinking the pulmonary data won't have much effect on what, 1)overall health, 2) influence on the AdCom panel 3) both? And in this case, increasing a single variable AND having a small data set, the "chance" factor is almost nil. Yet, the pulmonary data in Study 202 and the confirmatory study are robust, especially seen in older boys who are no longer ambulatory. You're taking a step in the right direction admitting you were wrong, like A.F. admitting he was wrong about Biomarin having sufficiently good enough data to impress AdCom panel members.
Also, it's nice to see that even though your past air of superiority in assessing prior data, and arrogant attitude towards posters who disagreed with you, is amenable to down-shift and a more conciliatory tone, evidenced by your public consumption of modest taste of humble pie.
"Of course dystrophin production is required to positively impact the disease butal its presences doesn't guarantee the health of the boys will improve."
Appears you are discounting the benefit of Etep on pulmonary function, and if that's not an improvement in health, please tell me then what you would consider an improvement on health to be.
The FDA has already signaled to SRPT that there will be a panel review. The only thing we don't know exactly is what the date will be b/c the date is "tentative".
Clearly, most analysts still give BMRN at least a slim or 50% chance of approval. Most don't get the difference between Drisa and Etep, and think they are "similar" in the MoA and chemistry. They haven't done their homework and they will continue to blather nonsense.
BMRN's briefing docs were meant to drive the panel discussion so that no positive spin could be done by BMRN that would support Drisa's approval. Mission accomplished.
SRPT, while still in the penalty box (by ignorant analysts) for the small-N issue, will have an additional 2 months of safety data in the confirmatory trials, which is huge. They have the goods on "D" data, the parent advocates at SRPT's AdCom will be passionately positive about Etep, the "anecdotal" evidence will be uniformly positive, and safety is pristine. In the end, there may not be a preponderance of evidence (due to small N issues), but there will clearly be sufficiently good enough evidence for AA/conditional approval.
The ignorant "analysts" will continue to support the possibility of Drisa approval until BMRN receives the CRL, for all the right reasons. SRPT's share price will rocket when that happens.
Sentiment: Strong Buy
After the briefing docs, and after the no confidence vote by the panel, the kicker was Dr. Farkas' comment about believing that if enough DMD boys were dosed with Drisa, there would be a patient death (due to severe AEs).
Comment from AF's live blog: "Again Adam, the FDA will reject drisa. They don't have legal authority to approve becuase no substantive evidence of effectiveness has been offered. Remember - FDA is a legal entity and that's what the law says. They won't open up a pandora's box of relying upon anything other than empiracally derived data."
Adam Feuerstein has the best one going on right now. Taking comments from folks following. Just saying'
I couldn't get in on the FDA web based portal: they are full. Govt. IT is pathetic
No official "vote" but nonetheless, a vote of confidence by way of recommendation (or absence thereof), expressed tone, level of concern for risk/benefit of a toxic drug being dosed in children for a very long time, where efficacy signals are virtually nonexistent.
Let's see, Brian's a "Senior" Biotech Analyst and on 1-Oct he offered a PT of $47 for SRPT
This is a summary of his most recent post:
"He still believes that Sarepta (NASDAQ: SRPT) has only a 35% chance of getting approval for eteplirsen but with the loss of a competitor, he believes it will still warrants a $50 share price by the end of 2016."
"The analyst believes FDA's criticism of drisapersen applies to BioMarin's entire DMD platform, as the phosphorothioate oligo class appears to only cause harm as a DMD therapy. Leading him to believe it will never make it to market but even if it does, the black box warning and precipitous drops in platelet count render it non-competitive."
"Looking through the drisapersen briefing documents, one of the more significant issues in the eteplirsen briefing documents will be related to protein quantification. Although eteplirsen seems to be producing dystrophin (in contrast to drisapersen), they are not confident that the quantity is going to be impressive to FDA."
"Our eteplirsen approval odds stand at 35%" while he has taken his drisapersen approval odds down virtually to zero."
I'm not sure if Brian's right and left brain are communicating fully. He thinks BMRN's toxic drug has zero chance of approval but only gives SRPT a 35% chance of FDA approval. Where in the world has Brian's brain been, regarding Sarepta's full-on gallop from behind to emerge as the clear winner in this race? And a $50 price target? OMG, as a "Senior" biotech analyst, it's time somebody put you out to pasture.
Yep, BMRN's $1 mil filing fee got sort of lit on fire with the scathing briefing docs. And their investment in Drisa was just greedy b. s. Toxic is as toxic does. Their share price after tomorrow should reflect that.
Well said, bf109gee! Your clear thinking and well spoken perspective have always been much appreciated on this board by many of us. CG, unfortunately didn't fully appreciate the nuance of interfacing with the FDA in a deferential manner, while still remaining a passionate advocate. Sadly, he was a victim of his own ego. When a relationship breaks down badly, sometimes there's no repair, and I think even he realized he needed to step aside. In the end, the process moved ahead without friction.
Biomarin, on the other hand, is the epitome of corporate greed and hubris. And yes, PF was used. Maybe PPMD will sue BMRN for providing misleading information to them; seems appropriate.
Ed Kaye's proclamation that Sarepta "will own the DMD space" was prescient but also based on his knowledge of how flawed RNA's platform was to treat DMD. How badly flawed is not evidence for all to see. How JJ's team can possibly advocate for approval of this hideous drug next Tues. is remarkable. Greed, pure and simple.
conrad: been long Srpt since deep in the AVII days. I've never sold a share throughout this roller coaster, but have added to my position when Srpt was sub $14 earlier this year. Only question now is whether they can stay independent. Would not be surprised to witness a bidding war for Srpt in '16. Gilead comes to mind as a suitor, or Amgn, Celg, Pfe, Nvs......the list is long. Cheers!