Dear SMA Community,
Yesterday was a disappointing and frustrating day for our community. We learned of two FDA approval delays for Scholar Rock’s apitegromab and Biogen’s high-dose nusinersen/Spinraza.
While there are many unknowns regarding timelines and next steps, the most encouraging news in all of this is that the issues named by the FDA are not related to the clinical efficacy or safety of the potential treatments themselves. This means that we are not looking at the need to repeat long and extensive clinical trials, which would have pushed progress back even further.
The most urgent question right now is how the FDA will determine the path forward. We are working with Scholar Rock, Biogen, and the FDA to understand how these issues can be resolved along with the expected timelines toward approvals. This could possibly be done with a focused re-submission update and a shorter re-review process (in the range of 3 to 5 months) or if the FDA will require a full re-submission and a longer re-review period (in the range of 9 to 12 months).
As we work toward these timeline answers, we are also committed to keeping you informed. We know our community has important questions, including:
- Will there be any impact on Scholar Rock's Expanded Access Program (EAP) or trial participants?
- Will this delay impact current Spinraza dosing?
- When is it realistic to expect approvals after this delay?
We will collect answers on these points and will share updates as soon as we have them. In addition, we invite you to submit any additional questions that you may have here. We will then prepare a comprehensive FAQ to address them directly.
While we are facing more uncertainty than we have in some time, I am absolutely certain of the strength of our community and that we will not stop until these issues are successfully resolved.
Thank you for your ongoing support, advocacy, and partnership. Together, we will continue moving forward.
Best,
Kenneth Hobby
President
Cure SMA