Greenwich Biosciences, a GW Pharmaceuticals Company, Presents New Data for EPIDIOLEX (cannabidiol) in Patients with Tuberous Sclerosis Complex (TSC) at the American Epilepsy Society Annual Meeting - Seite 2

“We are pleased to present these results which demonstrate the potential of EPIDIOLEX to reduce both focal and generalized seizures associated with TSC,” said Justin Gover, CEO, GW Pharmaceuticals. “This is promising news for patients and clinicians, and we remain committed to helping those suffering from seizures associated with this difficult-to-treat disease. We look forward to submitting an sNDA to the FDA soon, with the goal of expanding the EPIDIOLEX product label in 2020.”

The safety profile observed in the study was generally consistent with findings from previous studies. Adverse events (AEs) occurred in 93% of the 25 mg/kg/day group, 100% of the 50 mg/kg/day group and 95% of the placebo group. Both doses had an acceptable safety profile, with fewer AEs reported with 25 mg/kg/day than 50 mg/kg/day. The most common AEs were diarrhea, decreased appetite and somnolence. Eight patients on EPIDIOLEX 25 mg/kg/day, 10 on 50 mg/kg/day and 2 on placebo discontinued treatment due to an AE. Additionally, 13% of 25 mg/kg/day patients and 25% of 50 mg/kg/day patients experienced elevated liver enzymes; 79% of these patients were also taking the antiepileptic drug (AED) valproate. Elevations in ALT/AST resolved in all patients. There were no cases of Hy’s law observed and there were no deaths in the trial.

Study Design
The randomized, double-blind, placebo-controlled trial was conducted at 46 sites in six countries. A total of 224 people (aged 1 to 65) with a confirmed diagnosis of treatment-resistant TSC were randomized to receive either EPIDIOLEX 25 mg/kg/day (n=75), EPIDIOLEX 50 mg/kg/day (n=73), or placebo (n=76) for 16 weeks (4-week titration and 12-week maintenance phase), added to current AED treatment. The primary endpoint was percent change from baseline in TSC-associated focal and generalized seizure frequency for EPIDIOLEX vs placebo over the treatment period. Key secondary endpoints were ≥50% responder rate, percent reduction in total seizure frequency (including focal sensory and epileptic spasms), and subject/caregiver global impression of change (S/CGIC) in overall condition. On average, patients were taking three AEDs, having previously tried and discontinued four other AEDs. The most common concomitant AEDs in this trial were valproate (45%), vigabatrin (33%), levetiracetam (29%), and clobazam (27%).