Epidyolex Receives UK Approval to Treat Seizures From Tuberous Sclerosis
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has expanded the list of conditions Epidyolex, a CBD-based medication, can treat.
The cannabis-derived oral spray, which is known as Epidiolex in the US, can now be prescribed for seizures associated with tuberous sclerosis complex (TCS), a rare genetic condition that causes benign tumors to develop in different parts of the body.
The third indication
Developed by GW Pharmaceuticals (which is now a subsidiary of Jazz Pharmaceuticals), Epidyolex is a purified, oral form of CBD.
It was initially approved for use in the UK in September 2019, in conjunction with the anti-epileptic medication clobazam, as a treatment for seizures associated with the rare epilepsies Lennox Gastaut syndrome and Dravet syndrome in patients aged two years and older.
The latest approval for TCS marks the third indication Epidyolex is licensed to treat in the UK.
“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,” Chris Tovey, executive vice president at Jazz Pharmaceuticals, said in an announcement from Jazz Pharmaceuticals on August 10.
The MHRA’s approval was based on data from a phase three study, which found that 25 milligrams per kilogram per day (mg/kg/day) of Epidyolex helped reduce seizure frequency compared to both baseline CBD and a placebo.
The approval applies in England, Wales, and Scotland. The medication was previously approved for TCS patients in Northern Ireland in April 2021, when Epidyolex was further authorized in all 27 countries of the European Union, Norway, Iceland, and Liechtenstein.
In the US, Epidiolex was approved to treat TSC patients by the Food and Drug Administration (FDA) last August.
Following the latest approval in the UK, GW Pharmaceuticals has said that it will work with the country’s National Institute for Health and Care Excellence (NICE) to secure reimbursement for eligible patients.
Commenting on the MHRA approval, the chief executive of the charity Tuberous Sclerosis Association, Louise Fish, said “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.”
Typically diagnosed in childhood, TSC is estimated to affect between 3,700 and 11,000 people in the UK.