Hey, FDA!!!
I know I said I was taking a break from my blog, but this is important!
Ok, so, basically, the Ataxia community needs YOUR help with getting the FDA to review a potential Ataxia-specific medication ASAP.
We need YOU (hi! yes, you!) to read this post to learn about what is going on, complete the super quick 5-question survey at the end, and then share this info to get more people involved! Thanks!!!
Event Summary (taken directly from the NAF website):
“Troriluzole, an experimental drug being developed by Biohaven, completed Phase 3 clinical trials last year. While the primary endpoint assessing benefit across all Spinocerebellar Ataxia (SCA) types was not met, Biohaven believes the results show benefit among participants with SCA Type 3 (SCA3). In May of this year, Biohaven submitted a New Drug Application (NDA) for troriluzole to treat SCA3.
The U.S. Food and Drug Administration (FDA) recently issued a Refuse to File letter to Biohaven, which means that it did not accept the application and will not review the full data presented in the NDA.
Since this, the National Ataxia Foundation (NAF) has worked closely with regulatory experts, partner patient advocacy organizations, and Ataxia clinicians to learn more about why this happened, as well as explore precedents set by similar rare diseases that had potential treatments in similar situations.
Based on what we learned, we are very discouraged by FDA’s decision to Refuse to File the application for troriluzole and believe people with SCA3 deserve the opportunity to have FDA consider the data in full, despite the fact that the primary endpoint was not met in the study. We believe reviewing the data in its totality is consistent with FDA’s own policies for rare diseases like SCA3.”
Watch a really great video explanation here:
Read the complete NAF Statement Letter here:
NAF Calls on the FDA to Apply Regulatory Flexibility and Accept the NDA Filing for troriluzole
Andddddd here’s what you can do about it:
“NAF plans to deliver our statement to FDA in the near future. As part of this, we want to represent your voice to the FDA too! We want to ensure the FDA hears from individuals with Ataxia, their family members, friends, other care partners, clinicians, and more!
FDA needs to hear from the members of this community on key topics that may help the 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 survey questions are written about SCA3, we welcome feedback from individuals with all forms of Ataxia and other neurodegenerative diseases.”
> Please help our small Ataxia community be heard by completing this 5-question survey that will be presented to the FDA on August 21 to show them that we deserve a review of this potential treatment:
https://www.surveymonkey.com/r/9KWPTBR
My Personal Thoughts:
We know this isn’t some miracle Ataxia cure. We know this drug will not help everyone who takes it. We know this seems like an insignificant issue that only affects a very small number of people.
But, remember, the SCA community has NEVER experienced anything like this ever before. No SCA clinical trial has EVER made it this far. So then the FDA refusing to even look at it is really discouraging and heartbreaking. We’ve just been waiting for something, for anything, for so long.
So, please, sign the survey & share this post to help us show the FDA that this is important and LOTS OF PEOPLE (not only that one group of patients within this rare disease) CARE.
Thanks so much for helping spread the word!