GW Pharmaceuticals plc (Nasdaq: GWPH)  has submitted a Type II Variation Application to the European Medicines Agency (EMA) seeking approval of EPIDYOLEX, (cannabidiol) oral solution, for the treatment of seizures associated with Tuberous Sclerosis Complex. TSC is a rare genetic condition and a leading cause of genetic epilepsy. The stock was moving higher by 7% in early trading to sell at $82, not much higher than the stock’s 52-week low of $72.

“We look forward to working with the EMA to demonstrate GW’s cannabidiol oral solution’s potential in this new indication and hope to make this rigorously tested cannabis-based medicine available to a new group of patients through a potential approval in due course. If approved, this will be the third licensed indication for GW’s cannabidiol oral solution in Europe,” said Chris Tovey, GW’s Chief Operating Officer.

TSC Disease

TSC is a condition that causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys, and lungs, and in which epilepsy is the most common neurological feature. TSC is typically diagnosed in childhood. TSC often occurs in the first year of life with patients suffering from either 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 Type II Variation Application is based on data from a positive Phase 3 safety and efficacy study. The study met its primary endpoint with patients treated with GW’s cannabidiol oral solution 25 mg/kg/day experiencing a significantly greater reduction from baseline in TSC-associated seizures compared to placebo (49% vs 27%; p=0.0009). Results for the 50 mg/kg/day dose group were similar, with seizure reductions of 48% from baseline vs 26.5% for placebo (p=0.0018). 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.