GW wins European backing to fight rare genetic disease
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: “GW‘s decision to evaluate Epidiolex in patients with tuberous sclerosis is based on findings from the physician-led Epidiolex expanded access program where the results of this open-label use of Epidiolex in children with TS have been very encouraging.
“GW has successfully submitted regulatory applications for Epidiolex in both the US and Europe for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. TS represents a near term opportunity to expand the potential indications for Epidiolex and reflects GW’s ongoing commitment to addressing the needs of patients with highly treatment-resistant seizures.”
GW has already received orphan drug designation from the FDA for CBD in the treatment of TS.