In a recent press release, Jazz Pharmaceuticals announced the initiation of the new phase III trial to investigate the efficacy and safety of Epidiolex (cannabidiol), known as Epidyolex in Europe, in children and adolescents with EMAS.
The randomized, double-blind, placebo-controlled study will examine EMAS-associated seizure frequency over the 14-week treatment period compared with baseline. The company’s cannabidiol is not currently approved in the U.S. or European Union for the treatment of EMAS.
The phase III pivotal trial will be conducted in two parts and will enroll children and adolescent participants (aged 1-18 years) at 30 global sites. Part A will evaluate efficacy and safety of cannabidiol compared with 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 trial was launched based on preliminary data from the manufacturer’s clinical development program, including real-world evidence, that support cannabidiol as an effective therapy for the treatment of myoclonic atonic–associated seizures.
Rob Iannone, MD, MSCE, executive VP, global head of research and development of Jazz Pharmaceuticals, stated, “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. 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 content contained in this article is for informational purposes only. The content is not intended to be a substitute for professional advice. Reliance on any information provided in this article is solely at your own risk.
« Click here to return to Pediatrics Update.