Cholesterol 24-hydroxylase (CH24H) Inhibitor Soticlestat exerts its potential effects via the inhibition of Cholesterol 24-hydroxylase (CH24H). CH24H converts cholesterol to 24S-hydroxycholesterol (24HC) in the brain. Reduction of 24HC has been shown to reduce glutamatergic signaling and inflammation, which may have downstream effects on seizure susceptibility.
Populations Tested In
Developmental Epileptic Encephalopathies / Dravet syndrome and Lennox-Gastaut syndrome
Special FDA Designation
Rare Pediatric Disease
Nonprofit or Government Support
Nonprofit or Government Support Description
Presented at meetings (latest date) (Pipeline, AEDD; AES, Eilat, etc)