JAZZ PHARMACEUTICALS PLC Late-breaking presentations include a post-hoc analysis examining real-world outcomes of Epidiolex (cannabidiol) in treatment-resistant focal epilepsies within the Expanded Access Program (EAP), as well as a post-hoc analysis of the GWPCARE6 Open-Label Extension (OLE) trial evaluating the effectiveness of Epidiolex as add-on therapy against focal seizures in tuberous sclerosis complex (TSC). Two additional late-breaking abstracts will feature interim results from the BECOME-TSC (BEhavior, COgnition, and More with Epidiolex) Survey of caregivers of patients with TSC. These abstracts will report seizure and non-seizure outcomes with real-world use of Epidiolex in TSC and further expand the results of the original BECOME survey conducted among caregivers of people with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS) that were presented at the 2022 AES meeting.
'Multiple presentations at the 2023 AES meeting continue to build the scientific evidence in support of Epidiolex and improve understanding around the treatment's full impact,' said
Data highlights at the 2023 AES annual meeting include: A post-hoc analysis of patients diagnosed with treatment-resistant focal epilepsy within the EAP that demonstrated Epidiolex was associated with a sustained reduction in focal-onset seizures through 144 weeks. Notably, a second post-hoc analysis investigated the effectiveness and safety results of Epidiolex in adult patients from the EAP which found similar outcomes among patients with treatment-resistant epilepsies, including focal seizures. The EAP was an initiative launched in 2014 to provide add-on Epidiolex to patients with treatment-resistant epilepsy at 35 U.S. epilepsy centers.
A post-hoc analysis of the GWPCARE6 OLE trial evaluating the effectiveness of Epidiolex as add-on therapy against focal seizures in TSC. At least 50% reduction was reported by the majority of patients across focal seizure types through 144 weeks. Additionally, responder rates for focal seizure subtypes were consistent with the overall focal seizure responder rates, and the safety profile observed in the analysis was consistent with that observed in its overall clinical development program.
Interim results of the BECOME-TSC survey, in which the majority of caregivers reported patient improvements in overall seizure frequency and severity, as well as improvements in patients' cognition, emotional functioning, and communication domains.
About
Tuberous sclerosis complex (TSC) is a rare genetic condition.1 The condition causes mostly benign tumors to grow in vital organs of the body including the brain, skin, heart, eyes, kidneys and lungs2 and is a leading cause of genetic epilepsy.3 People with TSC may experience a variety of seizure types. One of the most common is infantile spasms that typically present in the first year of life; focal (or partial) seizures are also very common.4 TSC is associated with an increased risk of autism and intellectual disability5 and the severity of the condition can vary widely. In some children the disease is very mild, while others may experience life-threatening complications.4 Epilepsy is present in about 85 percent of patients with TSC and may progress to become intractable to medication.4,6,7 More than 60 percent of individuals with TSC do not achieve seizure control8 with standard treatments such as antiepileptic drugs, epilepsy surgery, ketogenic diet, or vagus nerve stimulation8 compared to 30-40 percent of individuals with epilepsy who do not have TSC who are drug resistant.
About Dravet Syndrome
Dravet syndrome (DS) is a rare genetic condition that appears during the first year of life with frequent fever-related seizures (febrile seizures). Later, other types of seizures typically arise, including myoclonic seizures (involuntary muscle spasms).11 Additionally, status epilepticus, a potentially life-threatening state of continuous seizure activity requiring emergency medical care, may occur. Children with DS typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others.
About Lennox-Gastaut Syndrome
Lennox-Gastaut syndrome (LGS) begins in childhood. It is characterized by multiple types of seizures. People with LGS begin having frequent seizures in early childhood, usually between ages 3 and 5.12 More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably. Almost all children with LGS develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with LGS require help with usual activities of daily living.
About Epidiolex/Epidyolex (cannabidiol)
EPIDIOLEX/EPIDYOLEX is a prescription, plant-derived cannabis-based medicine administered as an oral solution which contains highly purified cannabidiol (CBD). Cannabidiol, the active ingredient in EPIDIOLEX, is a cannabinoid that naturally occurs in the Cannabis sativa L. plant. The precise mechanisms by which EPIDIOLEX exerts its anticonvulsant effect in humans are unknown. Epidiolex, approved by the
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