GW Pharmaceuticals plc, a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, has announced that the European Medicines Agency (EMA) has granted orphan designation to GW's investigational product Epidiolex® (cannabidiol or CBD) in the treatment of Dravet syndrome, a rare and catastrophic treatment-resistant form of childhood epilepsy.
In addition to this orphan designation by the EMA, GW has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for Epidiolex in the treatment of Dravet syndrome as well as orphan designations in both Dravet syndrome and Lennox-Gastaut syndrome (LGS). GW is about to commence a full clinical development program for Epidiolex in the treatment of both Dravet syndrome and LGS, working with leading pediatric epilepsy specialists across the U.S. The first Phase 2/3 clinical trial is due to commence in the coming weeks.
On October 14th, GW announced updated physician reports of Epidiolex treatment effect in children and young adults with treatment-resistant epilepsy participating in open-label, "expanded access" studies. Of the 58 patients in this report, 12 patients had Dravet syndrome. These Dravet syndrome patients reported a median overall reduction in convulsive seizure frequency of 51%-72% across a range of time points and analyses. The most common adverse events were somnolence and fatigue.
"Dravet syndrome represents a very substantial unmet need in Europe and a significant therapeutic challenge as many of the children suffering with this condition are resistant to current treatments and have exhausted all options," stated Justin Gover, GW's Chief Executive Officer. "GW is now advancing a full clinical development program for Epidiolex in Dravet syndrome and looks forward to commencing this program in the coming weeks. We believe that the clinical effect and safety data recently released on Epidiolex support GW's confidence in the prospect of ultimately enabling children with Dravet syndrome around the world to have access to an approved prescription CBD medicine."
The EMA orphan designation is a status assigned to a medicine intended for use against a rare condition (prevalence of the condition in the European Union must not be more than 5 in 10,000) and allows a pharmaceutical company to benefit from incentives offered by the EU to develop a medicine for the treatment, prevention or diagnosis of a disease that is life-threatening or a chronically debilitating rare disease. These incentives can include reduced fees and protection from competition once the medicine is placed on the market.
About Dravet syndrome
Dravet syndrome, also known as Severe Myoclonic Epilepsy of Infancy (SMEI), is a rare and catastrophic form of epilepsy for which there is currently no cure. Seizures begin in the first year of life in an otherwise typically developing infant. Initial seizures are most often prolonged events (status epilepticus) and, in the second year of life, other seizure types emerge. All seizure types are remarkably resistant to medical therapy and the prognosis for Dravet syndrome is poor. Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy) and have associated co-morbid conditions, which also need to be properly managed. Children with Dravet syndrome do not outgrow this condition and it affects every aspect of their daily lives.