Yesterday, the FDA issued a statement identifying data accuracy issues with Avexis’ SMA therapy, Zolgensma. Cure SMA is in communication with the FDA and is committed to representing the SMA patient voice and ensuring it is represented in any and all discussions.
While the manipulation of data is a very serious matter, the agency has re-reviewed the clinical data and continues to believe that Zolgensma is safe and effective. We support the FDA’s decision and trust the agency to address the issue appropriately.
We have opened our own investigation into the matter, and we are looking forward to full transparency from Novartis and AveXis. As a first step, we have asked the company to provide a full report on whether the manipulated data ever was 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?
We will be issuing updates to the community on these topics as we get them. Meanwhile, we continue to urge families to consult their personal clinicians on the best course of treatment.
Based on the FDA’s evaluation that Zolgensma is still safe and effective and should remain on the market, Cure SMA will continue its efforts to advocate for broad insurance and site access for patients who are eligible. We remain engaged in dialogue with multiple health insurance providers to advocate for the broadest possible coverage within the FDA labels.
This issue of utmost importance and concern to Cure SMA and we will provide regular updates as new information becomes available.