The Only FDA-Approved CBD Treatment For Severe Epilepsy Completes Key Phase 3 Trial In Japan Despite Missed Primary Goal

Nina Zdinjak , Benzinga Staff Writer

August 23, 2024 9:15am Comments

The Only FDA-Approved CBD Treatment For Severe Epilepsy Completes Key Phase 3 Trial In Japan Despite Missed Primary Goal

Jazz Pharmaceuticals (NASDAQ:JAZZ) reported results from a Phase 3 trial in Japan evaluating the safety and efficacy of cannabidiol oral solution, Epidiolex, for treating seizures in children with rare forms of epilepsy, such as Lennox-Gastaut syndrome, Dravet syndrome and tuberous sclerosis complex.

While the trial did not achieve its primary goal of a significant reduction in seizure frequency, there were numeric improvements in other areas. Importantly, no new safety issues were found.

“We are confident in the overall clinical profile ofEpidiolex, which has been established in five Phase 3 clinical trials in more than 900 patients," stated Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "We believe the totality of the Epidiolexglobal data, including the findings from this trial, supports advancement of the program in Japan. We are continuing to collect data in Japanese patients and plan to engage with regulatory authorities in Japan regarding a potential new drug application (JNDA). We recognize the significant unmet need for patients in Japan living with rare epilepsies and thank the investigators, patients and caregivers who are involved in this trial.”

Epidiolex is the only FDA-approved prescription CBD treatment for severe forms of epilepsy. It was developed and produced by GW Pharmaceuticals, which was acquired by Jazz Pharmaceuticals in 2021. Epidiolex was first approved for treating seizures connected to Lennox-Gastaut syndrome and Dravet syndrome. In 2022, Epidiolex was approved for treating seizures related to tuberous sclerosis complex.

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The trial is focused on measuring how much the seizure frequency changes over a treatment period of up to 16 weeks in 62 children aged 1 to 18 years old. The trial is still ongoing to gather more data on how well the treatment works and its safety for both children and adults in Japan.