GW Pharmaceuticals receives orphan drug designation for cannabidiol treatment
GW Pharmaceuticals announced that the European Medicines Agency has granted orphan drug designation for cannabidiol for the treatment of tuberous sclerosis. GW is currently recruiting a Phase 3 clinical trial of Epidiolex as an adjunctive therapy for the treatment of seizures associated with TS, with data expected in the second half of 2018. Subject to positive results, GW expects to submit regulatory applications in 2019 for Epidiolex in TS in both the U.S. and Europe. TS is multisystem, genetic disease that causes benign tumors to grow in the brain and on other vital organs. TS can be life threatening in patients with severe symptoms, including drug resistant seizures and kidney failure. Up to 80 to 90% of individuals with TS will develop epilepsy during their lifetime, with onset typically in childhood. The seizures are often severe, and up to two-thirds of TS patients do not respond adequately to available medical therapies. There are significant co-morbidities associated with TS including cognitive impairment, autism spectrum disorders, developmental delay with severe learning disability and neurobehavioral disorders in individuals with TS. The EMA orphan designation is a status assigned to a medicine intended for use against a rare condition 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. GW has already received orphan drug designation from the FDA for CBD in the treatment of TS.