You may have seen that recently the United States Food and Drug Administration (FDA) issued a refuse to file letter to Biohaven regarding their New Drug Application (NDA) for troriluzole to treat Spinocerebellar Ataxia Type 3.
We are helping National Ataxia Foundation (NAF) with their effort to encourage the Food and Drug Administration to apply appropriate regulatory flexibility when reviewing New Drug Applications (NDA) for potential therapies for ataxia and we need your help.
We are concerned the FDA’s decision to Refuse to File the application may amount to a false negative conclusion, denying patients access to a drug that works. Only a full review of the data would give us confidence that this is not the case. We believe that FDA regulations and policies, as well as previous review of other drugs for rare diseases, support reviewing the troriluzole NDA.
We’re helping NAF with organising comments from the community to submit to the FDA. A full explanation and NAF’s statement are available on NAF’s website.
Please take 5 minutes to complete NAF’s survey to support the effort and provide your comment to the FDA. We want to ensure the FDA hears from people affected by ataxia, their family members, carers, clinicians, and more, since any decision FDA makes will impact you and your close ones. FDA needs to hear from the members of ataxia community on key topics that may help FDA understand why applying regulatory flexibility is warranted and why reviewing the full clinical trial data is in the best interest of patients.
While the questions are written about SCA3, feedback from all types of neurodegenerative diseases are important – from a personal & professional perspective. NAF will compile comments and them share with the FDA.
If preferred, you can submit an anonymous response.
We are looking for a strong response from the community – so please complete the survey and encourage others to as well. Share this with your friends. Thank you for your help in spreading the word!
We are looking for a strong response from the community – so please complete the survey by 21st August 2023 and encourage others to as well. Share this with your friends. Thank you for spreading the word!