GW Pharmaceuticals, a British pharmaceutical company discovering, developing and delivering regulatory approved cannabis-based medicines, has announced that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) approved a new indication for GW’s Epidyolex (cannabidiol) as an adjunctive treatment of seizures associated with tuberous sclerosis complex (TSC), for patients two years of age and older.
The company, which is now a part of Jazz Pharmaceuticals, was initially granted marketing authorization for this medicine in September 2019. It is 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.
GW has also received positive recommendations from the UK’s National Institute for Health and Care Excellence. This authorization for TSC represents the fourth approved indication of a 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 from 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, Jazz Pharmaceuticals’ executive vice president, chief operating office, and managing director. “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 manufactured here to even more patients across the UK. The authorization, 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.”
TSC is a condition that primarily causes benign tumors to grow in vital organs of the body, including the brain, skin, heart, eyes, kidneys, and lungs. Epilepsy is the most common neurological feature.
Estimated that between 3,700 and 11,000 people in the UK live with TSC, it is typically diagnosed in childhood.
In a statement, Tuberous Sclerosis Association (TSA) Chief Executive Louise Fish said, “One in every two people living with TSC-related epileptic seizures has difficulty treating 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 definitely need further options for clinicians who are managing seizures associated with TSC, and we therefore welcome this decision by the MHRA.”
Founded over two decades ago in response to significant unmet patient needs, GW’s mission is putting patients first and improving their quality of life. Their pioneering work has led to the regulatory approval of world first, potentially life changing, cannabis-based medicines.
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 percent in patients taking cannabidiol 25 mg/kg/day compared with 27 percent for placebo.
All key safety profile 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 program represents the only well-controlled clinical evaluation of a cannabinoid medication for patients with refractory epilepsy.
The approval from the MHRA applies in England, Wales, and Scotland. It also follows the recent approval of the product in all 27 countries of the European Union, alongside Norway, Iceland, Liechtenstein, and Northern Ireland. Following this approval, GW will work with the relevant stakeholders in the UK, including NICE, to secure reimbursement for eligible patients.
