- Clinical trials on using Epidyolex for TSC-related epilepsy are due to report in full in December 2019 at an epilepsy conference in America.
- We are expecting the company that make the drug to put in an application for a European license to use the drug in Q1 2020 (January, February or March).
- Once the drug receives a license, which should be later in 2020, NICE and the SMC will be asked to recommend whether the drug should be used on the NHS.
- We hope that they will make a decision before the end of 2020.
On Monday 11 November the BBC and other media outlets reported that two cannabis-based medicines, Epidyolex used to treat some rare forms of epilepsy and Sativex used to treat symptoms of multiple sclerosis, have been approved for use by the NHS in England. (https://www.bbc.co.uk/news/health-50351868).
Doctors will be able to prescribe Epidyolex for children with two types of severe epilepsy – Lennox Gastaut syndrome and Dravet syndrome – which can cause multiple seizures a day. It is estimated there are 3,000 people with Dravet and 5,000 with Lennox Gastaut syndrome in England. Epidyolex is already licensed for use in both of these conditions. Clinical trials showed the oral solution, which contains cannabidiol (CBD), could reduce the number of seizures by up to 40% in some children.
GW Pharmaceuticals – who make Epidyolex, a cannabidiol oral solution – have also reported positive phase 3 pivotal trial results in patients that have seizures associated with Tuberous Sclerosis Complex (TSC). The trial results for different dose groups were similar, with seizure reductions of 48.6% and 47.5% from baseline respectively, versus 26.5% for patients taking a placebo (dummy solution). The trial was conducted at more than 40 clinical sites in more than six countries.
GW Pharmaceuticals are planning to present their full phase 3 data on Epidyolex for TSC-related epilepsy to clinicians at the American Epilepsy Society annual meeting in December 2019, and to submit a license application to the European Medicines Agency (EMA) for TSC-related epilepsy in Q1 2020.
Once Epidyolex has received a license from the EMA to treat TSC-related epilepsy in 2020, the National Institute for Health and Care Excellence (NICE) will issue draft guidance on whether to recommend it for use by the NHS in England. Decisions on drug availability are devolved around the UK, but the NICE guidance should also apply in Wales and Northern Ireland. The Scottish Medicines Consortium (SMC) will consider whether to recommend the drug for use by the NHS in Scotland. We hope that final decisions will be made by all of the devolved nations during 2020.
The other drug approved for use by the NHS in England, Sativex, is licensed for treating some symptoms of Multiple Sclerosis. It is not being trialled for use in TSC.
All enquiries should be directed to the TSA’s Head of Support and Information Services, Rachael Wyartt:
Phone – 0300 222 5737
Email – Rachael.Wyartt@tuberous-sclerosis.org