SMA News Forums Forums Treatments and Research Evrysdi (risdiplam) FDA Approval for Risdiplam Delayed

  • FDA Approval for Risdiplam Delayed

    Posted by deann-r on April 8, 2020 at 9:23 am

    With so many of us anticipating risdiplam’s approval, it’s disappointing to learn it might not be approved until mid-August.  You can read more about it here. While this news isn’t what we want to hear there is a plus side. Because more trial information was included it’s feasible to believe once approved it could have an expanded label. Therefore may include a wide range of ages and types. Personally I was hoping approval would come sooner rather than later. What are your thoughts on this announcement?

    halsey-blocher replied 4 years, 2 months ago 4 Members · 3 Replies
  • 3 Replies
  • robert-stump

    April 8, 2020 at 1:56 pm

    How do I feel about?  SAD

    In regular times, sure, I understand, and yes I understand the FDA’s desire to have the latest data so they can make the most informed decision, but now in these times?  Times like these when the FDA is allowing off-label use of hydroxychloroquine just because he-whose-name-must-not-be-spoken says “what do you have to lose” even though no scientific data shows its effectiveness in treating COVID-19 yet?  Times like these when those with comorbidities, including SMA, need all the strength they can possibly muster when confronted with a respiratory illness like this?  Times like these when health-care rules are being rewritten on the fly, when HIPPA guidelines are ignored left and right, and when single-use N95 masks are being used for weeks on end and now “permissible” to be cleaned and reused 20 times?

    I don’t know, maybe I just can’t wrap my head around the fact that science and data still matter when it comes to whether I can get this life-saving drug now, or not…

  • lupa-f

    April 8, 2020 at 7:10 pm

    It does seem strange they’re doing this when Spinraza was given an expanded label despite having no trials for older patients at all. It might’ve been better if Roche actually gave them less data in this instance (though obviously that’s a bit less ethical).

  • halsey-blocher

    April 8, 2020 at 10:03 pm

    I have to admit I’m a bit disappointed. I’ve had my hope for receiving a treatment dashed once so I’ve been trying not to get my hopes up to much, but this is still a bummer. I understand why they’re doing it, but don’t really understand why they need quite that much time for it.

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