Replies to post #205628 on Biotech Values

[iwfal]

AVXS IONS BIIB

Why couldn't they enroll patients x-US (perhaps some x-EU too) where I presume Nusinersen would be later to market? Given EU reimbursement timelines they could potentially get the majority in Europe and not have to resort to other territories.

I'd be very surprised if, within 1 year, Nusinersen isn't approved in US and most of Europe. Further BIIB is pushing compassionate use so my guess most known cases of SMA Type 1 will be getting it well before then. . And, as I noted, even if they managed to enroll some it will be problematic to prevent them from taking Nusinersen. They really are in a tough spot, but a historical trial right now was probably their worst possible choice.

[rkrw]

Seem to be overlooking that Nusinersen is a chronically given intrathecal lumbar injection. With the strong early sentiments on efficacy, I doubt a one time gene therapy will have any trouble at all enrolling patients or have any issues with parents and their administering docs knowingly violating protocol with Nusinersen on top of the GT.