The Lancet has published data from a Phase III study backing use of GW Pharmaceutical/Greenwich Bioscience’s Epidiolex in patients with Lennox-Gastaut syndrome (LGS).
LGS is a rare, lifelong form of epilepsy associated with a high mortality rate and significant developmental delays. Patients suffer from multiple types of seizures, including drop seizures, which can result in falls and other injuries.
According to GW, results from The Lancet-published study “represent the only well-controlled clinical evaluation of a cannabinoid medication for this severe, drug-resistant condition.”
In the trial, 171 patients with LGS whose seizures were not controlled by their current AED regimen were randomised to receive either Epidiolex (cannabidiol) or placebo in addition to existing treatment. At baseline, patients had a median frequency of 74 drop seizures per month.
Over the 14-week treatment period (two-week dose escalation period followed by 12 weeks of maintenance), patients taking Epidiolex had a significantly greater median reduction in drop seizures compared to placebo (44 percent versus 22 percent), the study’s primary endpoint.
Sensitivity analyses confirmed that the treatment effect of CBD was established during the first month of treatment and was sustained over the entire treatment period, the group noted.
Key secondary goals were also met, showing that significantly more patients on cannabidiol experienced a 50 percent or greater reduction in drop seizures compared to placebo (44 percent vs 24 percent) and total seizure frequency was significantly reduced (median percent reduction of 41 percent vs 14 percent, respectively), while the drug was also considered to be well tolerated.
Also, CBD patients/caregivers were significantly more likely to report an improvement in overall condition with cannabidiol than placebo (58 percent vs 34 percent).
“Uncontrolled seizures significantly impact the lives of patients and their families and there is a tremendous need for new options in difficult-to-treat epilepsies such as LGS,” said Philip Gattone, president and chief executive of the Epilepsy Foundation.
“This randomised, controlled clinical study provides positive evidence of the potential role of cannabidiol in reducing seizures and we are excited about the possibility of a new treatment option for LGS.”
“Publication of this landmark study by The Lancet is an exciting achievement and marks the second time that Epidiolex data have been published in a highly prestigious journal, following last year’s publication in The New England Journal of Medicine,” said Justin Gover, GW's chief executive.
“We are absolutely focused on the goal of making this important potential new therapy available to appropriate patients and their caregivers as quickly as possible.”
Epidiolex is currently being reviewed by regulators on both sides of the Atlantic for LGS and Dravet syndrome, another difficult to treat childhood epilepsy.