GW Pharmaceuticals receives approval for Epidyolex to treat seizures

TSC causes epilepsy in up to 85 % of patients, and up to 60 % of those patients do not respond to standard anti-seizure medicines

GW Pharmaceuticals announces that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved a new indication for GW's cannabidiol as an adjunctive treatment of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older.

GW was initially granted marketing authorisation for this medicine in the UK in September 2019 as adjunctive therapy for seizures associated with Lennox Gastaut syndrome (LGS) or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. GW also received a positive recommendation from the UK's National Institute for Health and Care Excellence (NICE) for this medicine in November 2019 to receive routine reimbursement from NHS England. This authorisation for TSC represents the fourth approved indication of cannabis-based medicine in the UK for GW and underlines GW's commitment to the UK and regulatory approved cannabis-based medicines.

"We are delighted by today's decision from the MHRA for this new indication, which represents a significant step forward for TSC patients in the UK, many of whom may benefit from this new treatment," said Chris Tovey, Executive Vice President, Chief Operating Officer and Managing Director, Europe & International at Jazz Pharmaceuticals. "The UK is a significant country for us for many reasons, and we are proud to be able to offer medicine, that has been developed and is manufactured here, to even more patients across the UK. ‘’

The approval, made through the European Commission Decision Reliance Procedure (ECDRP), is based on data from a positive Phase 3 safety and efficacy study evaluating 25 mg/kg/day of GW's cannabidiol. The study met its primary endpoint, which was the reduction in seizure frequency compared to baseline of cannabidiol vs placebo, with seizure reduction of 49% in patients taking cannabidiol 25 mg/kg/day compared with 27% for placebo (p=0.0009). All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified. GW's development programme represents the only well-controlled clinical evaluation of a cannabinoid medication for patients with refractory epilepsy.

TSC is a condition that causes mostly benign tumours to grow in vital organs of the body, including the brain, skin, heart, eyes, kidneys and lungs, and in which epilepsy is the most common neurological feature. TSC is typically diagnosed in childhood. It is estimated that between 3,700 and 11,000 people in the UK live with TSC.