Two questions: does E work? If so when will it be licensed. The second is contingent on the first. The longer a handful of boys remain stable the more compelling the evidence is that E works. The nitty gritty statistics traditionally carry more weight when efficacy margins are thin, for example in cancer survival. But when a 100% fatal, progressive disease is arrested in all subjects, even in a small study, tradition has to be discounted in favor of direct observation. One factor that hasn't really been discussed here is the staffing levels at the FDA in early November. At the end of October and into the first 2 weeks of November 6500 FDA employees were furloughed. They were operating at about 50% staff levels, and there was no certainty about whether or when they would get to full strength again. I believe the posture of the FDA had more to do about their capacity to administer AA given the uncertainty of staffing levels and the resulting low morale than the prospects of the drug. But It would not have been politically correct to give the real reason for their vacillation.
that is very true... FDA said premature to file NDA... it turns out that not only was them saying that premature because they did not have relevant data in hand to make that assumption but we now have MORE reassurance of stability and over 2 years of safety
Premature was correct in the sense that the CMC was not submitted for review. If AA was/is to be considered SRPT must be able to supply GMP grade product immediately, and for a large % of the kids. There will be an immediate demand for approved supply. It is no longer a concern that the kids are not benefitted, what was addressed yesterday was that mid scale GMP material will be submitted to FDA in the first quarter. This is/was (past tense) a supply chain issue. When the supply chain submission goes to FDA it will no longer be '"premature at this time'. It will be time for kids with DMD to have 'available' supply of drug