EMA Grants Orphan Drug Designation to Epidiolex for Treatment of Lennox-Gastaut Syndrome

April 3, 2017
Pharmaceutical Technology Editors

GW Pharmaceuticals plans to submit a regulatory filing to FDA and EMA following two positive Phase III trials of Epidiolex in patients with Lennox-Gastaut Syndrome.

Biopharmaceutical company, GW Pharmaceuticals announced that the European Medicines Agency (EMA) has granted orphan drug designation to GW’s investigational product Epidiolex (cannabidiol [CBD]) in the treatment of Lennox-Gastaut Syndrome (LGS), a treatment-resistant, debilitating childhood-onset epilepsy.

"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," Justin Gover, the company’s CEO said in a press statement. According to Gover, GW is preparing to submit a new drug application with FDA in the middle of 2017, and to EMA shortly after that.

GW has also been granted orphan drug designation by EMA for Epidiolex in the treatment of Dravet syndrome. In the US, GW has received orphan drug designation from FDA for Epidiolex in the treatment of LGS, Dravet syndrome, tuberous sclerosis complex (TSC), and infantile spasms, each of which are severe infantile-onset, drug-resistant epilepsy syndromes. FDA has also granted fast-track designation to Epidiolex for the treatment of Dravet syndrome.

Epidiolex is GW’s lead cannabinoid product candidate. It is a proprietary oral solution of pure plant-derived CBD. GW is developing the compound for the treatment of severe, orphan, early-onset, treatment-resistant epilepsy syndromes including Dravet syndrome, LGS, TSC, and infantile spasms.

Source: GW Pharmaceuticals