A cannabis-based gel formulation developed by Zynerba Pharmaceuticals can significantly reduce seizures by up to six months in children and adolescents with developmental and epileptic encephalopathies, according to Phase 2 trial data.
Developmental and epileptic encephalopathies, or DEE, is an umbrella term that includes a heterogeneous group of rare pediatric epilepsy syndromes, such as Dravet and Lennox-Gastaut syndrome, among others.
The open-label BELIEVE 1 clinical trial was designed to explore the long-term safety and efficacy of Zygel in children and adolescents with DEE. To date, the study has enrolled, by invitation, 48 participants between 3 and 16 years old, who had confirmed DEE as classified by the International League Against Epilepsy.
The participants were initially dosed daily with 250 or 500 mg of Zygel, based on their weight, but the dosage could be increased up to a maximum of 1,000 mg daily. The treatment was directly applied to the skin (transdermal delivery) on the shoulders and upper arms.
Zygel was generally well-tolerated, and the safety profile was consistent with data from previous clinical studies.