Jazz Pharmaceuticals plc JAZZ announced new real-world data from the BECOME (BEhavior, COgnition, and More with Epidiolex) caregiver survey that showed a substantial proportion of caregivers of adult and pediatric patients with Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) prescribed Epidiolex (cannabidiol) oral solution reported improvements in patients' seizure and non-seizure outcomes across all age groups. These data were presented at the 2022 American Epilepsy Society annual meeting taking place December 2–6, 2022, in Nashville, TN.
Epidiolex, a pharmaceutical formulation of highly purified CBD, is the first and only prescription, plant-derived, cannabis-based medicine approved by the U.S. Food and Drug Administration. It is indicated for the treatment of seizures associated with LGS, DS, and tuberous sclerosis complex in patients aged one year and older.
"Severe, childhood-onset epilepsies like LGS and DS are lifelong conditions that have significant, long-term impacts on both caregivers and patients. These results are critical in broadening the knowledge base of the potential benefits of Epidiolex treatment across all age groups and identifying additional seizure and non-seizure outcomes to explore in future studies," stated lead author and investigator Timothy B. Saurer, medical affairs director of Jazz Pharmaceuticals. "We look forward to additional insights from the BECOME survey as we work to further characterize the specific effects of Epidiolex in both adult and pediatric patients living with rare, treatment-resistant epilepsy conditions."
Results
The results from both poster presentations suggest that a substantial proportion of the caregivers of people with LGS or DS treated with Epidiolex reported improvements in many patients' seizure and non-seizure outcomes, regardless of age group (patients aged <18 years and >18 years). Limitations to the survey included retrospective caregiver accounts, post-hoc analyses that were not pre-specified, and selection bias due to study design. In addition, the studies were not blinded and there were no comparator arms.
Seizure-Related Outcomes by Age
The seizure-related outcome analysis found a large majority of caregivers reported patient seizure improvements as measured by decreased frequency in at least one seizure type (84% aged <18 years and >18 years) and severity (77% aged <18 years; 75% in >18 years). Respondents reported patient improvements in specific seizure types: convulsive seizures (72% each), drop seizures (71% each), non-convulsive/non-drop seizures (66% aged <18 years; 69% in >18 years) and night-time seizures (61% and 63%).
Many respondents also reported reductions in use of rescue medications (57%), emergency room visits (56% and 51%), hospitalizations (55% and 50%) and occurrence of seizure-related injuries (48% and 49%).
Non-Seizure-Related Outcomes by Age
A substantial proportion of caregivers of patients with LGS or DS, regardless of age, reported improvements in alertness, cognition and executive function, emotional and social function, language and communication, physical function, sleep, and daily activities since initiating Epidiolex treatment.
A significant proportion of respondents in both age groups (patients aged <18 years and >18 years) reported any improvement (possible, minor, definite) in one or more questions regarding the following non-seizure outcome areas: Alertness, cognition, and executive function (87% and 81%) and emotional and social function (82% and 80%).
In patients aged <18 years, the most frequently reported improvement was the ability to learn new things (76%). For patients >18 years, the most frequently reported improvement was in alertness (70%).
Rates of worsening varied by behavior and ranged from 0% to 20% of respondents.
The most common side effects for Epidiolex include: sleepiness, decreased appetite, diarrhea, increase in liver enzymes, feeling very tired or weak, rash, sleep problems, fever, vomiting and infections.
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