The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved a new indication for GW Pharmaceutical’s Epidyolex cannabidiol as an adjunctive treatment of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older.
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. It’s thought that the condition affects up to 11,000 people in the UK, with the majority of these being diagnosed in children.
GW, now part of Jazz Pharmaceuticals, was initially granted marketing authorisation for Epidyolex in the UK in September 2019 as an 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.
The authorisation for TSC represents the fourth approved indication of a cannabis-based medicine in the UK for GW.
“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 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.”
Commenting on the MHRA approval of GW’s cannabidiol, 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 is based on data from a positive Phase 3 safety and efficacy study evaluating 25 mg/kg/day of GW’s cannabidiol. The study’s primary endpoint was the reduction in seizure frequency compared to baseline of cannabidiol vs placebo. The study found that 49% of patients had a reduction in seizures compared to 27% of the placebo group.