Biohaven announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for the New Drug Application (NDA) seeking approval of VYGLXIA (troriluzole) for the treatment of spinocerebellar ataxia (SCA).
A CRL means the FDA has completed its review and determined that the application cannot be approved in its current form. The CRL details the specific reasons why the application was not approved. It also outlines what the company must do to address those issues before potentially reapplying.
Read more in their press release.
NAF Statement
The FDA’s decision is disappointing for the Spinocerebellar Ataxia (SCA) community, who continue to wait for the first treatment for this devastating disease. For people living with SCA, time matters. Each day without treatment means more lost abilities and fewer options for maintaining quality of life.
We respect the FDA’s responsibility to ensure that treatments are safe and effective. At the same time, we believe that the experiences and priorities of people living with Ataxia must play a larger role in these decisions. Rare diseases simply must be reviewed more flexibly than more common conditions that have larger populations. When no approved treatments exist, regulatory flexibility and consideration of patient risk tolerance are not just important, they are essential.
As Vlad Coric, M.D., Chairman and Chief Executive Officer of Biohaven said, “The leading SCA experts in the United States directly communicated their support of the troriluzole data.” Unfortunately the Office of Neuroscience’s limited engagement with the patient community and leading experts leave us with concerns about the lack of regulatory flexibility that is being applied for rare, life-threatening conditions.
We call on the FDA to continue incorporating the patient experience as meaningful evidence in its evaluations and to apply regulatory flexibility in alignment with existing guidance for rare and progressive diseases. As the agency reviews additional data or considers future submissions, we urge a collaborative approach that recognizes both the unmet need and the voices of those directly affected.
NAF will continue working with our colleagues at Biohaven, the FDA, policymakers, and the Ataxia community to make sure the patient voice is represented in every stage of this process. While this moment is difficult, our commitment to the Ataxia community remains unwavering. We will keep pushing forward. Together, we can ensure that scientific progress is guided by compassion, urgency, and the lived experiences of those who live with this devastating disease.
