FDA Approval for Risdiplam Delayed

The importance of newborn screening for SMA

Rory and Carolyn met in seminary, fell in love, and got married. They decided to hold off on starting their own family while they settled into their first jobs in North Dakota. After a few years, they moved to Minnesota, where Rory accepted a job as a minister, and Carolyn enrolled in a hospital chaplain residency program. At the time, they did not know how important the decision to move would be for their future family. When they moved, neither North Dakota nor Minnesota had SMA on their newborn screening panel. It wasn’t until March 2018 that Minnesota adopted and implemented newborn screening for SMA, and then several months later, in 2018, Rory and Carolyn’s only son Edan was born.

Tagged: delays, FDA, FDA approval, risdiplam, SMA treatment

This topic has 3 replies, 3 voices, and was last updated 5 months, 2 weeks ago by Halsey Blocher.

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- April 8, 2020 at 9:23 am #23026
  
  DeAnn R
  Keymaster
  
  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?
- April 8, 2020 at 1:56 pm #23032
  
  Robert Stump
  Participant
  
  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…
- April 8, 2020 at 7:10 pm #23034
  
  Lupa F
  Participant
  
  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).
- April 8, 2020 at 10:03 pm #23036
  
  Halsey Blocher
  Participant
  
  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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