GW Pharmaceuticals plc, a Nasdaq-quoted biopharma business with operations in Cambridge UK, has won orphan drug approval from the European Medicines Agency (EMA) for a cannabis-based treatment of a rare genetic disease.
Its cannabidiol (CBD) solution treats tuberous sclerosis (TS) – a disease that causes benign tumours to grow in the brain and on other vital organs.
GW is currently recruiting 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 expects to submit regulatory applications in 2019 for Epidiolex in TS in both the US and Europe.
TS can be life threatening in patients with severe symptoms, including drug resistant seizures and kidney failure. Up to 80-90 per cent of individuals with TS will develop epilepsy during their lifetime, with onset typically in childhood.
CEO Justin Gover (pictured) said: