On February 27, 2018, GW Pharmaceuticals, PLC (Nasdaq:GWPH) (“GW) announced that the European Medicines Agency (EMA) has granted orphan drug designation for cannabidiol (CBD) for the treatment of tuberous sclerosis (TS).
GW has started a Phase 3 clinical trial of Epidiolex® (cannabidiol) 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 will submit regulatory applications in 2019 for Epidiolex in TS in both the U.S. and Europe.
Founded in 1998, GW is a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform in a broad range of disease areas.
The EMA orphan designation is a status assigned to a medicine intended for use against a rare, chronically debilitating, condition and allows a pharmaceutical company to benefit from incentives offered by the EU. These incentives can include reduced fees and protection from competition once the medicine is placed on the market. At the time of designation, tuberous sclerosis affected approximately 52,000 people in the European Union.
Tuberous sclerosis, also called tuberous sclerosis complex (TSC), is a rare, multi-system genetic disease that causes benign tumors to grow in the brain and on other vital organs such as the kidneys, heart, eyes, lungs, and skin. It usually affects the central nervous system and results in a combination of symptoms including seizures, developmental delay, behavioral problems, skin abnormalities, and kidney disease.