EC Grants Approval to GW Pharmaceuticals’ Epidyolex for TSC-Associated Seizures

Rare Daily Staff

The European Commission approved GW Pharmaceuticals’ Epidyolex as an adjunctive treatment of seizures associated with tuberous sclerosis complex, for patients two years of age and older. This approval, which represents the third indication for GW’s cannabidiol in Europe, paves the way for the expanded launch of the medicine across Europe.

Tuberous Sclerosis Complex (TSC) is a rare genetic condition that has an estimated prevalence in the European Union of 10 in 100,000. The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys, and lungs and is a leading cause of genetic epilepsy. The onset of epilepsy in TSC often occurs in the first year of life with patients initially suffering from predominantly focal seizures or infantile spasms. It is associated with an increased risk of autism and intellectual disability. The severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.

The approval is based on data from a positive phase 3 safety and efficacy study evaluating 25 mg/kg/day of GW’s Epidyolex. The study met its primary endpoint, which was the reduction in seizure frequency compared to baseline of cannabidiol vs placebo, with seizure reduction of 49 percent in patients taking cannabidiol 25 mg/kg/day compared with 27 percent for placebo. All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified.

GW’s Epidyolex was originally approved by the European Medicines Agency in September 2019 as an adjunctive therapy for seizures associated with Lennox-Gastaut syndrome or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older.

The EC decision is valid in all 27 countries of the European Union, alongside Norway, Iceland, and Liechtenstein.