Re: FDA filing
Thanks. That's another reason to delay the filing until "Fall". Probably the main one. We'd want to wait a bit to be able show the FDA some meaty data from a good chunk of Phase 3 kids in order to cement their decision. (And make it a safe one for them - what with all the squealing chihuahuas nipping at their ankles in one of their usual self-induced nervous Nellie frenzies whenever they see someone with balls doing something meaningful)
I think we started dosing after Thanksgiving 2022....It's 48 weeks....And we had a large group in line to get the shot at the start, so I'm guessing by year end this year we should have data on 30+ kids - the first two month's worth of dosed boys. If we file for AA October 1st, and we can give them final data on 30 kids by early February, they'll have almost 2 months (and possibly a few more kid's data) to make their decision by April 1st. That's probably the best scenario. e might file a bit later if we think we need more time to collect sufficient confirmatory phase 3 data.
On the PPMO trial for exon 51, we should hear soon on the restart of that once-a- month dose trial. It's been almost a month that it's been on hold and so it's possible most US kids will not have missed a dose as yet. (Ex-US no such interruption.) If some have had to skip a dose, they will represent a possibly interesting cohort if we see slightly differing data, but I doubt it'll make much difference. It could prove a plus if they end up benefitting from skipping a dose!