Jazz Pharma to trial Epidyolex as treatment for Doose syndrome

A new clinical trial will evaluate the potential of the CBD-based medicine, Epidyolex, in patients with Doose syndrome.

Jazz Pharmaceuticals has begun a new phase 3 trial to investigate the efficacy and safety of Epidyolex  in children and adolescents suffering from epilepsy with myoclonic-atonic seizures (EMAS), also known as Doose syndrome.

Epidyolex is a prescription, cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD).

Doose syndrome is a developmental and epileptic brain condition that begins in early childhood. The disease accounts for between one and two per cent of all childhood-onset epilepsies, according to a 2020 study. Seizures in children with Doose syndrome are often difficult to treat and may not respond well to medication.

The randomised, double-blind, placebo-controlled study will investigate EMAS-associated seizure frequency over the 14-week treatment period compared to baseline.

The phase 3 pivotal trial will be run in two parts and will enrol children and adolescent participants aged one to 18 across 30 global sites. The first part of the trial will assess the efficacy and safety of CBD compared to placebo. Participants will then have the option to continue in a 54-week open-label extension to.

The phase 3 trial was initiated based on preliminary data from Jazz Pharmaceutical’s clinical development programme, including real-world evidence, that supports cannabidiol as an effective therapy for the treatment of myoclonic-atonic-associated seizures.

Rob Iannone, executive vice president and global head of research and development at Jazz Pharmaceuticals, commented: “Given there are numerous treatment-resistant epilepsy syndromes, epileptologists often look for efficacy by seizure type, most of which have no syndrome-specific approved treatment.

“An EMAS indication would provide support for the use of Epidyolex in a fourth indication of a distinct, generalised seizure type, myoclonic-atonic seizures.”

He added: “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.”

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