GW Pharmaceuticals plc (GWPH) is one step closer to bringing its cannabidiol based drug, Epidiolex, to market. On Jan. 24, 2017, the company announced, along with its U.S. subsidiary Greenwich Biosciences, that The Lancet has published results from a Phase 3 study of Epidiolex in patients with Lennox-Gastaut syndrome (LGS).
The study’s publication marks the first-ever well-controlled clinical study of cannabidiol in LGS. GW Pharmaceuticals has submitted a New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for Epidiolex, which was approved in December with an assigned PDUFA goal date of June 27, 2018. If approved, Epidiolex could find its way to U.S. markets by the second half of 2018. GW Pharmaceutical stock was up slightly on the news and was trading at $138 a share.
LGS is a rare form of epilepsy that manifests itself during childhood and last throughout the patient’s lifetime. Often associated with a high mortality rate and developmental delays, LGS patients can be afflicted with multiple types of seizures, many of which can result in serious injuries.
In the study, patients treated with Epidiolex experienced a significant drop in monthly seizure frequencies compared to those treated with a placebo. Treatment of Epidiolex was generally well tolerated, with few adverse effects.
The randomized study was conducted with 171 patients suffering from LGS. ranging from age 2 to 55. Approximately half of the patients (85) were given a placebo while the other half (86) were given Epidiolex.
Over the course of the 14 week treatment period, patients treated with Epidiolex experienced a significantly greater average reduction in seizures (44%) compared to those treated with a placebo (24%). Epidiolex patients were also more likely to report an improvement in their condition (58%) than those treated with a placebo (34%).
Approximately 86% of patients receiving Epidiolex reported adverse side effects, compared to 66% of patients receiving a placebo, but most effects were mild and well tolerated. The most common side effects reported were diarrhea, drowsiness, elevated body temperature, decreased appetite, and vomiting.
“The publication of these positive results is an exciting milestone for the LGS community and we are encouraged that a new treatment option could soon be available,” said Christina SanInocencio, executive director of the Lennox-Gastaut Syndrome Foundation, in a statement. “Additional treatment options are desperately needed for patients who continue to struggle with uncontrolled seizures and these results offer much-needed hope to those living with this debilitating condition.”