Epygenix Therapeutics received an orphan drug designation from the FDA for its EPX-300 treatment for patients with Dravet Syndrome.
The syndrome is a lifelong form of epilepsy that qualifies as a rare pediatric disease. The company discovered the drug’s effectiveness using phenotype screening.
With the latest designation, all of Epygenix’s drug candidates have received orphan status for Dravet Syndrome. The company says it is now equipped to develop “a diversified pipeline” of treatments for the disease.