-12 Patients without Primary end point review (Efficacy) just in Phase 2 data more on Safety does give US FDA an indication for approval.
Common US FDA is not stupid - Guidelines/Pathways are issued all companies who puts the pressure on FDA from bribed small community voices, does not prove efficacy..
GSK failed so as many other similar drugs.
I feel petty on those analyst who raises price target without even having NDA filled, that to be promised by end of 2014 and approval by 2015.
FDA is allowing [Sarepta] to start a confirmatory eteplirsen study with an open-label design, although we are cautious that the FDA is also encouraging the company to start placebo-controlled studies for other exon-skippers as soon as possible in order to build the case for eteplirsen approval. We are cautious that the FDA will have the same concerns that were highlighted in today’s press release when it reviews eteplirsen in 2015, and may opt to continue to wait for more controlled data to be convinced. Importantly, the FDA believes it is not appropriate to exclude patients who lose ambulation while on drug from the analysis (modified intent-to- treat), which has been key for eteplirsen to compare favorably to historical control (which includes such patients). The FDA has shown a willingness to be patient and wait for more compelling/controlled data before approving eteplirsen so we see the risk that the FDA continues to do so beyond the first filing at year-end.
Late last year, the agency suggested that the company needed to do a larger, late-stage study for eteplirsen, which treats Duchenne muscular dystrophy treatment, a fatal disease that destroys muscle tissue in children and afflicts about one in every 3,500 boys born across the globe, according to Sarepta.
Analyst common ...NDA to be in 2014 end Approval in 2015 if control study is done ....1/3500 might have MDD where eteplirsen can be approved but GSK failed ......UPGRADE TODAY !!!!!!!!!!!!!
Understand that....and FDA give this to every company not just SRPT.
END OF 2014 APPLICATION TO BE FILLED BASED ON WHAT? 12 Patients data in Phase 2 which company claimed as initially satisfactory but not proved yet...!! How about FDA guidelines which require to conduct a Blinded trials and prove efficacy by an independent data monitoring committee.
12 Patients are contacted and advised to be pretending Very good with this drug until Dec 2014.
Food and Drug Administration telling the company last November that it had concerns about granting an accelerated approval for Sarepta's muscular dystrophy drug eteplirsen.
The decision wasn't a big surprise after Prosensa (NASDAQ: RNA ) and GlaxoSmithKline's (NYSE: GSK ) related drug, drisapersen, failed its phase 3 trial last year.
12 Patients in a private trial considered as Phase2 without GSK knows worked very well. FDA had already typed approval just remained to be faxed to CEO. Adam also paid very well to keep quite until signal comes, All analyst were delivered cash and options shares so that they shall be ready before PR to issue Price Tgt raised within few Hrs, Everything in place but we believe the news only marginally de-risks the program. Given the side effect profile observed thus far, we would be surprised if safety issues arose that might derail approval
12 patients privately treated and results are excellent -CEO believed it and FDA agreed. Now next straight approval through OBAMA CARE .
January 13, 2014 |
GlaxoSmithKline ($GSK) has shut the door on its hopes in Duchenne muscular dystrophy (DMD), handing back rare-disease drug drisapersen to Prosensa ($RNA), its former partner.
The once-promising drisapersen works by inducing exon skipping, basically convincing the body to ignore the faulty dystrophin proteins that cause DMD. That promise came tumbling down in September, however, when the drug failed to beat out placebo in improving walking distance in patients with the muscle-destroying disease, sending Prosensa's shares into a 70% tailspin.
Now, as GSK washes its hands of the breakthrough-designated drug, the Netherlands-headquartered Prosensa is planning to go it alone on a salvage project, heading back to the drawing board with a handful of candidates
FDA meeting ...Promise to file NDA by end of the year, clinical trials (blinded), Paid to Adam to keep quite, Analyst paid in advance to raise Tgt , RESULT STOCK OFFERING ...$100Million with additional $15Million
FDA is still concerned about the small sample size of the mid-stage study and a primary endpoint has yet to be agreed upon for the review. Sarepta has lobbied the FDA to accept levels of dystrophin expression as a primary endpoint, but the agency has seemed to lean more toward results from the walking test. Put another way, the FDA wants to know that increased dystrophin production means something in terms of increasing a DMD patient's ability to function. And therein lies the problem.