Earlier this week, the FDA issued a statement identifying data accuracy issues with AveXis’ SMA therapy, Zolgensma. Cure SMA immediately opened an investigation into the matter and is committed to representing the SMA patient voice and ensuring it is represented in any and all discussions with the FDA, Novartis, and AveXis.

Below is an update of where we stand with our investigation, as well as what you can expect in the coming weeks:

  • Cure SMA has requested a report from AveXis on whether the manipulated data was ever presented to families and patients, Cure SMA staff or board members, or in grant applications and reports submitted to Cure SMA, or in any formal journal publications or presentations at conferences.
  • We are also seeking answers to the following questions:
    • What data was manipulated?
    • What is the real data?
    • When, where and why did this manipulation take place?
  • Today, AveXis released a community statement. You can read the full text of that statement here.
  • Next week, Cure SMA will announce a webinar to be held the week of August 19th. We hope to discuss this issue in more detail during this webinar and answer any questions from the community.

In the meantime, we trust the FDA’s evaluation from their statement that Zolgensma is safe and effective and should remain on the market. Cure SMA urges families to consult their personal clinicians on the best course of treatment and will continue its efforts to advocate for broad insurance and site access for patients who are eligible.

We want to reiterate that this issue is of the utmost importance and concern to Cure SMA. Our plan is to provide regular updates and communicate any new information as it becomes available.