The FDA, the US regulatory agency for new medicines, declined to review the documents for a potential therapy for SCA3 (type of ataxia) developed by Biohaven. The criteria for approving a drug often rely on standards established for more prevalent conditions, making it exceedingly challenging for rare conditions to fulfill certain FDA prerequisites.
In response, the National Ataxia Foundation (NAF) has initiated a campaign aimed at providing feedback to the FDA, advocating for the incorporation of regulatory flexibility in the evaluation of new drug applications for rare conditions like SCA3. This initiative holds significance for the DRPLA community as well. If the FDA were to adopt a more adaptable approach to assessing drug applications for rare conditions, it could potentially benefit the DRPLA community in the future.
A full explanation and NAF’s statement are available on NAF’s website.
We support NAF in making sure that the FDA hears from the ataxia community. NAF has put together a survey to gather all the feedback for the FDA. While the questions are written about SCA3, feedback from all types of neurodenerative conditions are important.
Make your voice heard! Fill out NAF’s brief survey by August 21, 2023 to have your feedback included in their outreach to the FDA.