07/30/2025
SLC6A1 Connect recently sponsored a clinical trial testing , an existing FDA‑approved drug, in children with SLC6A1 mutations. This is part of a broader wave of drug repurposing, where scientific curiosity and serendipity are unlocking new possibilities in epilepsy treatment.
Drug repurposing offers a faster, more cost‑effective path than traditional drug development, which often takes over a decade and billions in R&D. Phenylbutyrate—Ravicti’s active ingredient—has already shown promising seizure reduction in preliminary real‑world and clinical cases for SLC6A1, STXBP1, and other monogenic epilepsies.
Collaboration across researchers, clinicians, and patient advocacy groups like SLC6A1 Connect has fueled this progress—and it's making a real difference.
Yet, Ravicti remains prohibitively expensive (around $900K/year), and regulatory pathways for repurposed indications are still decisively incomplete. But for many families, especially those awaiting gene therapies, this trial offers a lifeline today, backed by hope and the possibility of meaningful improvement.
There’s much to be done—but the spark is very real.
Amber Freed. Mike Graglia Monica Elnekaveh Agustina Fernández Giambruno Laura Kababick Bermingham Rachel Forest Heilmann David Fajgenbaum
Repurposing currently FDA-approved drugs fast-tracks treatments for epilepsy, including the treatment of rare forms of epilepsy.
