London, UK-based GW Pharmaceuticals’ cannabinoid Epidiolex has been awarded Orphan status by European regulators as a treatment for Lennox-Gastaut Syndrome (LGS), a rare and severe form of childhood-onset epilepsy.
The move follows data from two late-stage trials showing that when Epidiolex was added as an adjunct to patients' current treatment, there was a significant reduction in the monthly frequency of drop seizures assessed over the entire 14-week treatment period versus placebo.
Drop seizures were defined as atonic, tonic and tonic-clonic seizures involving the entire body, trunk or head that led or could have led to a fall, injury, slumping in a chair or hitting the patient's head on a surface. During the treatment period, patients taking Epidiolex achieved a median reduction in monthly drop seizures of 44 percent compared with a reduction of 22 percent in patients receiving placebo.
On the safety side, the drug was generally well tolerated, the firm noted, with 86 percent of all Epidiolex patients experiencing an adverse event compared with 69 percent in the placebo arm, the most common of which were diarrhoea, somnolence, decreased appetite, fever and vomiting.
“Following two positive Phase III trials of Epidiolex in patients with LGS, GW is committed to pursuing registration of Epidiolex in Europe in order to provide these patients access to an approved prescription CBD medicine," said Justin Gover, the firm’s chief executive.
He confirmed that the company is preparing to submit an application to market the drug in the US for this indication in the middle of 2017, followed by a submission to the European Medicines Agency shortly after.