Jazz Pharmaceuticals plc (Nasdaq: JAZZ) has announced the top-line results from a Phase 3 open-label, single-arm trial conducted in Japan. The study assessed the safety and efficacy of their cannabidiol oral solution, marketed globally as Epidiolex®/Epidyolex®, as an adjunctive treatment for seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC). The trial did not achieve its primary efficacy endpoint, which was a pre-specified percentage change in seizure frequency related to these conditions during the treatment period (up to 16 weeks) compared to baseline in Japanese pediatric patients. Despite this, the study did show numeric improvements in both the primary and several secondary endpoints. Importantly, no new safety concerns were identified during the trial.
Rob Iannone, M.D., M.S.C.E., Executive Vice President and Global Head of Research and Development at Jazz Pharmaceuticals, expressed confidence in the overall clinical profile of Epidyolex. He highlighted that the drug’s efficacy and safety have been demonstrated in five Phase 3 clinical trials involving over 900 patients. Iannone emphasized that the collective data from these trials, including the findings from the recent Japanese study, supports the continuation of the program in Japan. Jazz Pharmaceuticals plans to engage with regulatory authorities in Japan regarding a potential new drug application (JNDA). Iannone also acknowledged the significant unmet needs of Japanese patients living with rare forms of epilepsy and expressed gratitude to the investigators, patients, and caregivers who participated in the trial.
About the Phase 3 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.
About Cannabidiol
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.
