Jazz Pharmaceuticals plc (NASDAQ:JAZZ) has initiated a new Phase 3 trial to investigate the efficacy and safety of Epidiolex (cannabidiol), known as Epidyolex in Europe, in children and adolescents with epilepsy with myoclonic-atonic seizures (EMAS). The randomized, double-blind, placebo-controlled study will investigate EMAS-associated seizure frequency over the 14-week treatment period compared to baseline. The company's cannabidiol is not currently approved in the United States or European Union for the treatment of EMAS.
EMAS – also known as myoclonic astatic epilepsy (MAE) or Doose syndrome – is a developmental and epileptic encephalopathy that begins in early childhood. EMAS accounts for between one and two percent of all childhood-onset epilepsies. Seizures in children with EMAS are often difficult to treat and may not respond well to medication.
"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 Epidiolex in a fourth indication of a distinct, generalized seizure type, myoclonic-atonic seizures," stated Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of 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 broad range of difficult-to-treat seizure types."
The Phase 3 pivotal trialmwill be run in two parts and will enroll children and adolescent participants (ages 1-18) at 30 global sites. Part A will assess 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 an option to continue in a 54-week open-label extension Part B.
The Phase 3 trial was initiated based on preliminary data from our clinical development program, including real-world evidence, that supports cannabidiol as an effective therapy for the treatment of myoclonic-atonic-associated seizures.
About EPIDIOLEX /EPIDYOLEX
EPIDIOLEX /EPIDYOLEX (cannabidiol), is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified CBD. Cannabidiol, the active ingredient in EPIDIOLEX, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which EPIDIOLEX exerts its anticonvulsant effect in humans are unknown.
