GW Pharmaceuticals says Phase III trials for Epidiolex to treat rare epilepsy in children shows positive results

pharmafile | June 27, 2016 | News story | Research and Development, Sales and Marketing Epidiolex, GW Pharmaceuticals, NDA, US FDA, drug trial, orphan drug 

GW Pharmaceuticals (Nasdaq: GWPH) said late-stage trials for its Epidiolex (cannabidiol or CBD) to treat Lennox-Gastaut syndrome (LGS), a rare and severe form of childhood-onset epilepsy achieved its primary endpoint. 

Epidiolex in combination with the current treatment showed a significant reduction in the monthly frequency of drop seizures assessed over the entire 14-week treatment period, the company said in a statement. The trial follows the positive results from a Phase III study in March for Epidiolex to treat Dravet syndrome. 

Chief Executive Justin Gover said: “We now look forward to advancing Epidiolex towards the submission of an NDA with the FDA in the first half of 2017.” 

Epidiolex has Orphan Drug Designation from the US Food and Drug Administration (FDA) for the treatment of LGS and Dravet syndrome. 

Gover, said: “We are delighted to announce positive results in this Phase III trial of Epidiolex in patients with Lennox-Gastaut syndrome, and particularly pleased that this result is consistent with our recent Phase III pivotal data for Epidiolex in Dravet syndrome. We believe that this result further demonstrates that Epidiolex offers the potential to be a new effective therapy within the field of treatment-resistant childhood-onset epilepsies.” 

Epidiolex, GW’s lead cannabinoid product candidate, is an oral pharmaceutical formulation of pure CBD, which is in development for the treatment of a number of rare childhood-onset epilepsy disorders. 

The peak onset of LGS typically occurs between ages of 3 to 5 years and can be caused by a number of conditions, including brain malformations, severe head injuries, central nervous system infections, and inherited degenerative or metabolic conditions. In up to 30% of patients, no cause can be found. It is estimated that there are approximately 14,000-18,500 patients with LGS in the US and 23,000-31,000 patients with LGS in Europe. 

Anjali Shukla

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