The New England Journal of Medicine has published the second of two late stage studies showing that GW Pharma’s cannabinoid Epidiolex cut seizures in patients with the rare childhood epilepsy Lennox-Gastaut Syndrome.
In the trial - the first dose-ranging study to compare cannabidiol to placebo as add-on therapy in LGS - both doses of Epidiolex significantly reduced drop seizure frequency in patients with poor seizure control despite the use of multiple anti-epileptic drugs.
According to the data, during the 14-week treatment period median reductions in monthly drop seizures were 37.2 percent (10 mg/kg/day dose) and 41.9 percent (20 mg/kg/day), compared with a 17.2 percent reduction for placebo.
Also of note, key secondary endpoints showed a significant number of patients receiving cannabidiol oral solution 10 mg/kg/day (36 percent) and cannabidiol oral solution 20 mg/kg/day (39 percent) experienced a 50 percent or greater reduction in monthly drop seizures compared with those taking placebo (14 percent).
Patients/caregivers were also significantly more likely to report an improvement in overall condition with both cannabidiol oral solution doses compared to placebo based on the Subject/Caregiver Global Impression of Change questionnaire, the firm noted.
"Positive study results, such as these, offer much needed hope for patients and their families living with this debilitating condition," said Christina SanInocencio, executive director of the Lennox-Gastaut Syndrome Foundation.
“New options are desperately needed and the LGS community is very excited about the potential of cannabidiol as a new, differentiated treatment option for patients for whom current treatments have failed to provide adequate benefit.”
The first Phase III trial assessing Epidiolex in LGS was published by The Lancet in January, which showed that over the 14-week treatment period, patients taking the drug had a significantly greater median reduction in drop seizures compared to placebo (44 percent versus 22 percent).
“We are now in the latter stages of the FDA’s review of our New Drug Application and look forward to a decision from FDA in late June. If approved, we expect to make this important potential new medicine available to US patients with LGS in the second half of the year,” said GW’s chief executive Justin Gover.