FDA Patient Listening Session

In March of 2026, the Cute Syndrome Foundation hosted a Patient Listening Session with the U.S. Food and Drug Administration (FDA) to share the real-life experiences of individuals and families impacted by SCN8A-related disorders. The session brought together patients, caregivers, clinicians, and advocates to help FDA representatives better understand the realities of SCN8A: not just the seizures, but the developmental, behavioral, physical, emotional, and day-to-day challenges that affect the entire family.

Throughout the session, we emphasized three things: current treatments are not enough, meaningful progress looks different for every person, and the need for better treatment options is urgent. Families shared their stories of treatment journeys, loss, uncertainty, and hope, while highlighting the need for treatments that can improve more than seizure counts, reduce medication side effects, and preserve development and quality of life. We also asked the FDA to consider the unique challenges of ultra-rare disorders like SCN8A when evaluating new treatments, including more flexible clinical trial approaches and meaningful measures of benefit beyond seizures.

Across the session, families emphasized that SCN8A is a whole-life, whole-family disorder. Seizures are urgent and often life-threatening, but the disease burden also includes developmental delay or regression, impaired communication, movement and muscle tone differences, feeding and respiratory vulnerability, sleep disruption, behavioral crises, caregiver vigilance, sibling impact, and grief. Participants repeatedly described a need for therapies that are more precise, less burdensome, and capable of improving daily functioning and quality of life—even when progress is incremental.

Potential areas for FDA consideration include: recognition that seizure count alone may not capture treatment benefit; inclusion of non-seizure endpoints and caregiver-observed functional changes; trial designs that reduce travel, placebo exposure, and participation burden; thoughtful risk-benefit assessment in a population already exposed to substantial medication and disease-related risk; and flexibility for very small, genetically defined subgroups.