The phase 3 trials will assess Epidiolex over 14 weeks in a randomised, double-blind, placebo-controlled study, enrolling paediatric patients aged one to 18. It is hoped that Epidiolex will ease symptoms associated with Doose syndrome (EMAS), a rarer type of childhood-onset epilepsy, accounting for 1-2% of all cases.
Epidiolex, developed by GW Pharmaceuticals, is an epilepsy treatment derived from cannabidiol (CBD), a chemical component of cannabis. However, it doesn’t include tetrahydrocannabinol (THC), the psychoactive compound cannabis is more well known for.
Epidiolex was the first cannabis-based drug therapy to be approved by the Food and Drug Adminstration (FDA) in 2018. It required a reclassification by the US Drug Enforcement Agency (DEA) before it could be commercialised.
EMAS tends to have a higher occurrence in boys than in girls and is linked to different types of seizures which include drop, tonic and myoclonic seizures. Jazz Pharma saw that seizures in paediatric EMAS patients were difficult to treat, with some children reacting badly to currently-recommended medications.
The trial will be split into two parts – the first will assess the efficacy and safety of cannabidiol compared to placebo as a treatment for adolescents with myoclonic-atonic seizures. The second will be an optional 54-week open-label extension trial.
Rob Iannone, executive vice president and global head of research and development of Jazz Pharmaceuticals, said: “Jazz is committed to continuing to generate clinical study data and real-world evidence to further support the utility of the company’s cannabidiol across a broad range of difficult-to-treat seizure types.”
