Jazz's CBD therapy fails in Japanese Phase III trial

Jazz Pharmaceuticals has encountered a hurdle in its quest to secure approval for its cannabidiol therapy in Japan. A Phase III clinical trial conducted in Japan to evaluate the therapy did not meet its primary goal. The trial tested the efficacy of the cannabidiol oral solution as an additional treatment for seizures related to Lennox-Gastaut syndrome (LGS), Dravet syndrome (DS), or tuberous sclerosis complex (TSC) in children. Unfortunately, the study did not achieve the pre-specified percentage change in seizure frequency over a 16-week treatment period compared to the baseline.

The cannabidiol therapy, known as Epidiolex in the United States and Epidyolex in the European Union, is derived from cannabis plants. Jazz Pharmaceuticals acquired this therapy as part of its 2021 purchase of GW Pharmaceuticals. The U.S. Food and Drug Administration (FDA) has already approved Epidiolex for treating seizures in pediatric patients with LGS, DS, and TSC, three rare forms of epilepsy.

Epidiolex/Epidyolex is a significant revenue driver for Jazz Pharmaceuticals, generating approximately $845.5 million in sales last year. According to financial forecasts by GlobalData, the drug's sales are expected to continue rising, potentially surpassing $1.3 billion by 2030.

Despite the disappointing results of the Phase III trial, Jazz Pharmaceuticals is determined to move forward with the development of the therapy in Japan. Rob Iannone, the company's executive vice president, expressed confidence in the overall clinical profile of Epidyolex. He emphasized that the therapy has been validated through five Phase III clinical trials involving more than 900 patients globally.

Iannone stated, "We are confident in the overall clinical profile of Epidyolex, which has been established in five Phase III clinical trials in more than 900 patients. We believe the totality of the Epidyolex global data, including the findings from this trial, supports the 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)."

The open-label Phase III trial in Japan involved 62 pediatric patients aged between 1 and 18 years, all of whom suffered from seizures due to either LGS, DS, or TSC. Although the primary endpoint was not met, Jazz Pharmaceuticals observed numerical improvements in both the primary and several secondary endpoints.

Jazz Pharmaceuticals remains committed to advancing the therapy and believes that the comprehensive global data on Epidyolex will support its regulatory journey in Japan. The company plans to continue gathering data from Japanese patients and discuss the potential for a new drug application with local regulatory authorities.

In summary, while the Phase III trial in Japan did not meet its primary endpoint, the numerical improvements observed and the robust global clinical data provide a basis for Jazz Pharmaceuticals to continue its efforts to introduce the therapy in Japan. The company remains optimistic about the future of Epidiolex/Epidyolex and its potential to benefit patients with rare epileptic disorders.

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