Jazz Pharmaceuticals to Present Epidiolex® Data at European Epilepsy Congress 2024

Jazz Pharmaceuticals plc has announced the presentation of eight abstracts at the 15th European Epilepsy Congress (EEC) held in Rome, Italy, from September 7-11, 2024. Among these presentations, key findings from the BECOME Caregiver Survey highlight the real-world impacts of Epidyolex® (cannabidiol) treatment on patients with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS).

The BECOME (BEhavior, COgnition and More with Epidyolex®) Caregiver Survey involved caregivers of patients with LGS or DS who reported significant improvements in both seizure and non-seizure outcomes for patients receiving Epidiolex/Epidyolex along with clobazam. The data showed that most caregivers observed improvements in seizure frequency, seizure severity, and weekly seizure-free days. Additionally, many caregivers reported positive changes in non-seizure-related domains such as alertness, cognition, executive function, language, and communication. An overwhelming majority of caregivers indicated their intention to continue with the Epidyolex treatment.

In a subgroup analysis of the BECOME survey, it was observed that treatment with Epidyolex and concomitant clobazam for a duration of at least three months led to significant improvements. The analysis included both pediatric (aged 2-17 years) and adult (aged 18 years and older) patients. Specifically, 87% of caregivers reported a reduction in seizure frequency, 81% noted a decrease in seizure severity, and 68% observed an increase in weekly seizure-free days. In non-seizure-related outcomes, 84% of caregivers saw improvements in areas like alertness and cognition, while communication and language skills improved in both verbal and non-verbal patients.

Another major highlight is the post-hoc analysis of data from the GWPCARE3 and GWPCARE4 Phase 3 trials, which involved 215 LGS patients treated with Epidyolex for 14 weeks. This analysis identified thresholds for clinically significant reductions in drop seizures. Out of the patients analyzed, 31% reported a 'much improved or better' Caregiver Global Impression of Change (CGIC) score, and 60% noted a 'slightly improved or better' CGIC score. The identified thresholds for a clinically important response were reductions in drop seizures of approximately 30.6% for a 'slightly improved or better' score and 49.6% for a 'much improved or better' score.

Further insights come from two analyses of real-world data from the Adelphi LGS and DS Disease-Specific Programmes™. These analyses underscore the challenges in diagnosing LGS and DS correctly. A significant proportion of patients were initially misdiagnosed with other seizure types before being correctly identified as having LGS or DS. The studies also revealed that most patients required multiple anti-seizure medications, highlighting the necessity for effective early treatment options.

Additionally, a late-breaking abstract highlighted the effects of a ketogenic diet (KD) on cannabidiol plasma concentration and seizure reduction in patients treated with Epidyolex in conjunction with clobazam. The post-hoc analysis of data from three randomized controlled trials found no substantial differences in cannabidiol plasma concentration or seizure responses between patients on a ketogenic diet and those who were not. Further research is needed to validate these findings.

Epidiolex®/Epidyolex® is a prescription, plant-derived cannabis-based medication approved in both the United States and the European Union for the treatment of seizures associated with LGS, DS, and Tuberous Sclerosis Complex (TSC) in specified age groups. The medication has shown promise in improving the quality of life for patients and their caregivers by addressing both seizure-related and non-seizure-related symptoms.

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