Jazz Pharmaceuticals, a leading pharmaceutical company, has reported results from a Phase 3 clinical trial in Japan evaluating the safety and efficacy of Epidiolex, a cannabidiol oral solution, for treating seizures in children with rare forms of epilepsy. The trial focused on children aged 1 to 18 years old with Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex.
While the trial did not achieve its primary goal of demonstrating a significant reduction in seizure frequency, there were notable numeric improvements in other areas. Importantly, no new safety concerns emerged during the trial.
Rob Iannone, executive vice president and global head of research and development at Jazz Pharmaceuticals, stated, “We are confident in the overall clinical profile of Epidiolex, which has been established in five Phase 3 clinical trials in more than 900 patients. We believe the totality of the Epidiolex global data, including the findings from this trial, supports advancement of the program in Japan.”
The company plans to continue gathering data on the efficacy and safety of Epidiolex in Japanese patients and intends to engage with regulatory authorities in Japan regarding a potential new drug application (JNDA). Jazz Pharmaceuticals recognizes the significant unmet need for patients in Japan living with rare epilepsies and expresses gratitude to the investigators, patients, and caregivers who participated in the trial.
Epidiolex is the only FDA-approved prescription CBD treatment for severe forms of epilepsy. Originally developed by GW Pharmaceuticals, which was acquired by Jazz Pharmaceuticals in 2021, Epidiolex was initially approved for treating seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. In 2022, its approval was expanded to include seizures related to tuberous sclerosis complex.
The trial in Japan, still ongoing, aims to collect more data on the treatment’s effectiveness and safety for both children and adults. Jazz Pharmaceuticals’ shares are currently trading down 4.36% on Friday morning.
This research is a testament to the ongoing efforts to find effective and safe treatments for patients suffering from rare forms of epilepsy. With the collection of further data and potential regulatory approval, Epidiolex could offer a significant treatment option for patients in Japan and beyond.
