Jazz Pharmaceuticals plc JAZZ has initiated a new phase 3 trial to investigate the effectiveness and safety of Epidiolex(cannabidiol), known as Epidyolex in Europe, in children and adolescents with eepilepsy with myooclonic-atonic sconvulsions (EMAS). The randomized, double-blind, placebo-controlled study will investigate the frequency of seizures associated with EMAS during the 14-week treatment period compared to baseline. The company’s cannabidiol is not currently approved in the United States or the European Union for EMAS treatment.
EMAS, also known as astatic myoclonic epilepsy (MAE) or Doose syndrome, is an epileptic and developmental encephalopathy that begins in early childhood. EMAS accounts for one to two percent of all childhood-onset epilepsies. Seizures in children with EMAS are often difficult to treat and may not respond well to medication.
“Because there are numerous treatment-resistant epilepsy syndromes, epileptologists often look for efficacy by seizure type, most of which have no syndrome-specific approved treatments. generalized seizure type, myoclonic seizures- sluggish,” he said Rob Iannone, MD, MSCE, Executive Vice President, Global Head of Research and Development at Jazz Pharmaceuticals. “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 wide range of difficult-to-treat seizure types.”
The pivotal Phase 3 trial will be conducted in two parts and will enroll child and adolescent participants (aged 1-18 years) at 30 global sites. Part A will evaluate the efficacy and safety of cannabidiol compared to placebo as an adjunctive treatment for children and adolescents with myoclonic-atonic seizures. Upon completion of Part A, participants will have the option to continue in an open 54-week extension to Part B.
The Phase 3 trial was initiated based on preliminary data from our clinical development program, including real-world evidence, supporting cannabidiol as an effective therapy for the treatment of myoclonic-atonic seizures .
About EPIDIOLEX /EPIDIOLEX
EPIDIOLEX / EPIDYOLEX (cannabidiol), is a plant-derived cannabis-based prescription drug that is administered as an oral solution containing highly purified CBD. Cannabidiol, the active ingredient in EPIDIOLEX, is a cannabinoid found naturally in the plant Cannabis sativa L. The precise mechanisms by which EPIDIOLEX exerts its anticonvulsant effect in humans are unknown.