Jazz Pharmaceuticals has announced top-line results from the Phase 3 open-label, single-arm trial in Japan evaluating the safety and efficacy of cannabidiol oral solution as an adjunctive treatment for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS) or tuberous sclerosis complex (TSC). The trial did not meet the primary efficacy endpoint of a pre-specified percentage change in indication-associated seizure frequency during the treatment period (up to 16 weeks) compared to baseline in Japanese pediatric patients; however, numeric improvements were observed in the primary and several secondary endpoints. No new safety signals were observed in the trial.
The Phase 3 open-label, single-arm clinical trial investigates the safety and efficacy of cannabidiol oral solution (GWP42003-P) for the treatment of seizures associated with LGS, DS or TSC in Japanese pediatric patients. The trial includes a pre-specified primary efficacy outcome measuring the percentage change in indication-associated seizure frequency during the treatment period (up to 16 weeks) compared to baseline in 62 patients ≥ 1 to ≤ 18 years of age. The trial design includes a treatment period consisting of an initial 2-week titration period followed by a 14-week maintenance period; and a period evaluating safety for up to 52 weeks of treatment. The trial remains ongoing to collect efficacy and safety data in pediatric and adult Japanese patients.
Cannabidiol, 100 mg/mL oral solution, a prescription, plant-derived cannabis-based medicine approved by the U.S. Food and Drug Administration (FDA) for use in the U.S., and the European Medicines Agency (EMA) for use in the European Union, is an oral solution which contains highly purified cannabidiol (CBD). In the U.S., cannabidiol, under the tradename Epidiolex, is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome or tuberous sclerosis complex (TSC) in patients one year of age and older. Cannabidiol has also received approval in the European Union, under the tradename Epidyolex, for adjunctive use in conjunction with clobazam to treat seizures associated with LGS and Dravet syndrome in patients two years and older, and for adjunctive use to treat seizures associated with TSC, in patients two years of age and older
