The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has approved a new indication for GW Pharmaceutical’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 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.
“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, made through the European Commission Decision Reliance Procedure (ECDRP), is based on data from a positive Phase III 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.5 It is estimated that between 3,700 and 11,000 people in the UK live with TSC.1
The approval from the MHRA applies in England, Wales and Scotland and follows the recent approval of the product in all 27 countries of the European Union, alongside Norway, Iceland and Liechtenstein (including Northern Ireland).
- Tuberous Sclerosis Association website. https://tuberous-sclerosis.org/information-and-support/what-is-tsc/. Accessed August 2021
- Nabbout R, Belousova E, Benedik MP, et al. Epilepsy in tuberous sclerosis complex: Findings from the TOSCA Study. Epilepsia Open. 2019 Mar; 4(1): 73–84.
- Boston Children’s Hospital. https://www.childrenshospital.org/conditions-and-treatments/conditions/t/tuberous-sclerosis-tsc/symptoms-and-causes. Accessed March 2021.
- Chu-Shore CJ, Major P, Camposano S, Muzykewicz D, Thiele EA. The natural history of epilepsy in tuberous sclerosis complex. Epilepsia 2010;51(7):1236–41.
- NIH Tuberous Sclerosis Fact Sheet. https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Tuberous-Sclerosis-Fact-Sheet.
- Prevalence and incidence of rare diseases: Bibliographic data. https://www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf
- TS Alliance Website. https://www.tsalliance.org/. Accessed August 2021
- de Vries PJ, Belousova E, Benedik MP, et al. TSC-associated neuropsychiatric disorders (TAND): findings from the TOSCA natural history study. Orphanet J Rare Dis. 2018;13(1):157.