Risk of no treatment: extreme; risk of etiplirsen: zero. 6MW test and dystrophin results: superb.Mandate for approval asap: quite obvious
SRPT's story in a nutshell.
No competing treatment approach.
FDA rapid approval, by whatever name, is begging for etiplirsen.
FDA's Temple has said even a non-surrogate, clinical endpoint can be used for Accelerated approval: 6MW results are stellar. Both he and Dr. Woodcock have alluded to small sample sizes like SRPT's being sufficient for approval.
30 patient years of safety have been the precedent for FDA approval in the past for similar disease categories: etiplirsen had 30 patient years of safety data as of August 2013.
Twitter this post headline if you want to convey the real bottom line SRPT story in a nutshell.
Yes, I agree.
And when Accelerated approval occurs, the clinical validation trial is called 'confirmatory' rather than phase III.
One can not do a rapid approval and at the same time wait for a phase III trial to occur.
The whole point of a confirmatory trial (after accelerated approval) is that the boys who would otherwise be going downhill into wheelchairs will be getting the drug while the confirmatory trial is proceeding. To wait months or years for a phase III to be completed is to needlessly sentence those boys to a wheelchair.
Etiplirsen is safe. Over 30 patient-years worth--enough for full approval of other drugs in the past.
But you cannot also do an accelerated approval with a required confirmatory trial, without starting the confirmatory trial first - they would never be able to recruit patients. This is one reason CG is pushing ahead to start the confirmatory trial in Q1. Beyond the Etep approval, this may be the best chance to recruit a large number of patients to generate a solid dystrophin database, which will help the cause with future Exons.
No way man! You are dreaming......all the statements from the recent SRPT meeting points to getting ready for phase III trial to prove if Eti is a fake or truly effective in a multi center prospectively designed pivotal randomized trial...SRPT knows it and the FDA knows it.......they are just not coming out in the open.