GW Pharmaceuticals has received approval for Epidyolex from the Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of seizures associated with tuberous sclerosis complex.
Epidyolex, the first cannabis-derived medicine to receive a positive NICE recommendation for the treatment of seizures in patients with two rare and severe forms of childhood-onset epilepsy, has now received MHRA approval for the treatment of seizures associated with tuberous sclerosis complex (TSC).
Tuberous Sclerosis Association (TSA) chief executive, Louise Fish, said: “One in every two people living with TSC-related epileptic seizures has difficult to treat epilepsy that does not respond to traditional anti-epileptic drugs.
“We are excited to see this new medicine approved by the MHRA, which gives people living with TSC and their families hope. We desperately need further options for clinicians who are managing seizures associated with TSC, and we, therefore, welcome this decision by the MHRA.”
The approval applies in England, Wales and Scotland and follows the recent approval of the product in all 27 countries of the European Union. Following this approval, GW Pharmaceuticals will work with the relevant stakeholders in the UK, including NICE, to secure reimbursement for eligible patients.
Tuberous sclerosis treatment
TSC, which causes mostly benign tumours to grow in vital organs of the body, affects an estimated 3,700 to 11,000 people in the UK, and causes epilepsy in up to 85% of patients – 60% of which do not respond to standard anti-seizure medications.
The approval is based on data from a positive Phase 3 safety and efficacy study evaluating 25 mg/kg/day of Epidyolex, which met its primary endpoint of the reduction in seizure frequency compared to baseline of cannabidiol vs placebo. Seizure reduction was 49% in patients taking cannabidiol 25 mg/kg/day compared with 27% for placebo.
Speaking on Tuesday, Chris Tovey, executive vice president, chief operating officer and managing director, Europe and international at Jazz Pharmaceuticals, commented: “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.
“The UK is a significant country for us for many reasons, and we are proud to be able to offer a medicine, that has been developed and is manufactured here, to even more patients across the UK. This authorisation, expanding the label, is further proof of our continued commitment to the UK and the pioneering research and development into regulatory approved cannabis-based medicines we have conducted here.”
GW Pharmaceuticals, which is now part of Jazz Pharmaceuticals, was initially granted marketing authorisation for the medicine in the UK in 2019 as an adjunctive therapy for seizures associated with Lennox Gastaut syndrome or Dravet syndrome, in conjunction with clobazam, for patients two years of age and older. The medicine also received a positive recommendation from NICE to receive routine reimbursement from NHS England.
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