Jazz Pharma Updates Phase 3 Japan Trial of Cannabidiol for Resistant Epilepsies

DUBLIN, Aug. 22, 2024 — Jazz Pharmaceuticals plc (Nasdaq: JAZZ) recently revealed top-line findings from a Phase 3 open-label, single-arm study in Japan. The study evaluated the safety and effectiveness of their cannabidiol oral solution, marketed globally as Epidiolex®/Epidyolex®, as an additional treatment for seizures linked to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC).

The trial's primary objective, which was a pre-specified percentage change in seizure frequency during the treatment phase (up to 16 weeks) compared to baseline in Japanese pediatric patients, was not met. Despite this, numerical improvements were noted in both the primary and several secondary endpoints. Importantly, no new safety concerns were identified throughout 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 Epidyolex’s overall clinical profile, which has been confirmed in five Phase 3 clinical trials involving over 900 patients. Iannone emphasized that the collective global data on Epidyolex, including this recent trial's findings, support advancing the program in Japan. He highlighted the ongoing data collection efforts and the company's intention to engage with Japanese regulatory bodies concerning a potential new drug application. Iannone also acknowledged the significant unmet needs of patients in Japan with rare epilepsies and thanked the trial's investigators, patients, and caregivers.

The Phase 3 study focuses on assessing the safety and efficacy of cannabidiol oral solution (GWP42003-P) for treating seizures associated with LGS, DS, or TSC in Japanese pediatric patients. The trial's primary efficacy outcome involves measuring the percentage change in seizure frequency during the treatment period, which includes a 2-week titration phase followed by a 14-week maintenance phase. The study encompasses 62 patients ranging from 1 to 18 years of age and includes a safety evaluation period lasting up to 52 weeks. The trial is still ongoing to gather more efficacy and safety data in both pediatric and adult Japanese patients.

Cannabidiol is a plant-derived cannabis-based medicine approved in the U.S. and the European Union. The oral solution, known as Epidiolex in the U.S. and Epidyolex in the European Union, contains highly purified cannabidiol (CBD). In the U.S., it is approved for treating seizures associated with LGS, DS, or TSC in patients aged one year and older. In the European Union, Epidyolex is approved for use alongside clobazam to treat seizures associated with LGS and DS in patients aged two years and older and for treating seizures associated with TSC in the same age group.

Jazz Pharmaceuticals plc is a global biopharmaceutical company committed to developing life-changing medicines for patients with serious diseases, often with limited treatment options. The company boasts a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, as well as an expanding range of cancer treatments. Headquartered in Dublin, Ireland, Jazz Pharmaceuticals operates research and development laboratories, manufacturing facilities, and employs a global workforce dedicated to serving patients worldwide.