SCA3 FDA Patient-Led Listening Session Summary

On September 22, 2023, the FDA hosted a listening session with advocates from the Spinocerebellar Ataxia Type 3 (SCA3) disease community. The National Ataxia Foundation and our partner organizations led efforts to invite SCA3 community members to share their experiences about living with the rare disease.

Session Objectives

To educate FDA staff on the complex issues, variety of physical manifestations, and body
systems affected by this rare disorder.
To educate FDA staff on the serious impacts of SCA3 disease manifestations on patients and
their quality of life.
To educate FDA staff on the scarcity of currently available treatments, tremendous unmet
medical need, and preferences for future treatments and outcomes.

Topics Discussed

An overview of SCA3, including the cause of disease, disease progression, symptomology, and
the impact on families and caregivers.
The symptoms and health effects most burdensome to people with SCA3.
The inadequacy of current treatments and unmet medical needs of patients with SCA3.