Results of a survey relating to the use of Epidiolex (cannabidiol) oral solution in patients with Lennox-Gastaut Syndrome (LGS) and Dravet Syndrome (DS) indicates improvement in patient outcomes.
Dravet and Lennox-Gastaut syndromes are rare conditions appearing during the first year of life, characterised by frequent fever-related seizures. These seizures tend to increase in frequency with age, and children typically experience poor development of various skills among other issues.
One of the treatments for managing the condition is Epidiolex, which is based on highly purified cannabidiol (CBD). It’s the only prescription, plant-derived, cannabis-based medicine approved by the U.S. Food and Drug Administration for the treatment of seizures associated with LGS, DS, and tuberous sclerosis complex (TSC).
Epidiolex, which is marketed under the name Epidyolex in Australia, was approved for the treatment of seizures associated with LGS or Dravet syndrome in patients two years of age and older in Australia in 2020.
It’s a very expensive medication, but some top-line results from a caregiver survey on its use are encouraging. A substantial proportion of the caregivers of people with LGS or DS who were treated with Epidiolex reported improvements in many patients’ seizure and non-seizure outcomes – and this wasn’t dependent on patient age.
In terms of seizures, a large majority of caregivers reported patient seizure improvements as measured by decreased frequency in at least one seizure type. In terms of specific seizure type observed improvements:
- convulsive seizures: 72% each age bracket – <18 years and >18 years
- drop seizures: 71% each
- non-convulsive/non-drop seizures: 66% aged <18 years; 69% in >18 years
- night-time seizures: 61% and 63%.
With regard to non-seizure related positive outcomes (possible, minor, definite improvements):
- alertness, cognition, and executive function: 87% and 81%
- emotional and social function: 82% and 80%)
Among patients aged less than 18 years, the most frequently reported improvement was the ability to learn new things (76%). For those 18 or older, the most frequently reported improvement was in alertness 70%.
“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,” said lead author and investigator Timothy B. Saurer, who is the Medical Affairs Director of Jazz Pharmaceuticals.
Jazz Pharmaceuticals acquired Epidiolex manufacturer GW Pharmaceuticals back in 2021, and integrated the firm into Jazz.
More detail and results from the Epidiolex caregiver survey can be found here.