New Data Supports Use Of Epidiolex In Tuberous Sclerosis Complex

Research shared at the American Epilepsy Society (AES) 2024 Annual Meeting held from 6-10 December in Los Angeles, California supports the use of Epidiolex in the treatment of rare epilepsies, including tuberous sclerosis complex (TSC).

TSC is a rare genetic condition that causes non-cancerous  tumours to develop in different parts of the body, often in the brain, skin, kidneys, heart, eyes and lungs. It can lead to other significant health problems, including epilepsy.

Epidiolex is approved for the treatment of seizures associated with Lennox-Gastaut syndrome, Dravet syndrome and TSC in patients one year of age and older.

The first data from the EpiCom trial, a prospective, interventional trial evaluating the impact of adjunctive Epidiolex on TSC-associated neuropsychiatric disorders (TAND), revealed improvements in behavioral symptom severity following treatment.

Additionally, analysis of real-world data from the BECOME-TSC (BEhavior, COgnition, and More with Epidiolex) caregiver survey and the BECOME-LTC survey, which evaluated the perspectives of nurses who care for patients with epilepsy in long-term care (LTC) facilities and group homes, reports improvements in seizure frequency and in certain non-seizure outcomes associated with Epidiolex treatment.

Two updated analyses of real-world outcomes from the BECOME-TSC study showed that of 55 caregivers who completed the survey, 89% planned to continue Epidiolex treatment for their loved one. 

The most important stated reasons for continuing Epidiolex included seizure and non-seizure benefits such as reduced seizure frequency and severity and duration, as well as TAND-related improvements in cognition and language and communication.

Results from the BECOME-LTC survey found that, among 102 nurses surveyed, 85% reported a reduction in overall frequency of any seizure type after Epidiolex initiation, with 49% reporting a greater than 50% reduction.

Improvements were also observed across different seizure subtypes as well as in non-seizure outcomes, with nurses reporting improvements in emotional functioning, sleep, cognitive abilities, ability to communicate, and physical functioning.

Results from CARE-EpiC (Caregiver Analysis of Real-world Epidiolex in Epilepsy Context), a cross-sectional caregiver survey, demonstrated reduced caregivers’ need for additional support of their dependents’ physical, emotional, and behavioral care after Epidiolex initiation and characterized improvements in their dependents’ well-being as well as caregivers’ experiences.

A subgroup analysis evaluating treatment outcomes in patients with TSC (TSC group) versus other types of focal epilepsy treated with Epidiolex in the U.S. Expanded Access Program, found Epidiolex has similar effectiveness in TSC and other focal epilepsies, regardless of focal epilepsy type.

In the TSC group, Epidiolex was associated with an average reduction of 51%–87% in focal seizures and 44%–87% in total seizures. In the non-TSC group, Epidiolex was also associated with a reduction of 46%–75% in focal and 46%–74% in total seizures.

Dr Sarah Akerman, Head of Neuroscience Global Medical and Scientific Affairs of Jazz Pharmaceuticals, commented: “Our real-world data presentations at AES 2024, including novel findings from the BECOME-LTC, BECOME-TSC and EpiCom studies, demonstrate the meaningful impact of Epidiolex in the treatment of patients with rare epilepsies.

“These findings increase our understanding of Epidiolex’s benefits beyond seizure control, addressing unmet needs across a range of epilepsy syndromes for people living with rare epilepsies and demonstrating reproducibility and consistency of effect across different populations.”