GW Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for cannabidiol (CBD) for the treatment of Tuberous Sclerosis Complex (TSC).
TSC is a rare genetic disorder, the most common symptom of which is epilepsy. Epilepsy occurs in around 80-90% of TSC patients and is a significant cause of morbidity and mortality. TSC is the third orphan indication that GW is targeting within its Epidiolex® (CBD) clinical development program, which includes four Phase 3 pivotal trials in Dravet syndrome and Lennox-Gastaut syndrome, both rare and catastrophic forms of childhood-onset epilepsy.
On April 11, 2016, GW announced it has commenced a Phase 3 clinical trial of Epidiolex® as an adjunctive therapy for the treatment of seizures associated with TSC.
