Zogenix Announces New Data on Psychological and Socioeconomic Impact of Dravet Syndrome and Other Epileptic Encephalopathies at 13th European Congress on Epileptology
Psychological Impact of Epileptic Encephalopathies on Siblings
Two presentations analyzed data from the
- The first analysis found that siblings of children with epileptic encephalopathies may be at risk for anxiety or depression, with a significant proportion of both younger siblings and adult siblings reporting symptoms of anxiety and depressed mood. These findings were presented in a podium session and nominated for an ECE Best Poster Award.
- A separate analysis of data from the
Sibling Voices Surveyfocused on the specific concerns of younger children (< 18 years old) who experience growing up with a sibling with an epileptic encephalopathy. A substantial percentage of siblings reported being worried or scared, having less attention from their parents, activities being disrupted, and more responsibilities. The most frequent emotions reported by the younger cohort (9 – 12 years old) included being easily startled, unhappiness and grumpiness, while the older cohort (13 – 17 years old) reported irritability, unhappiness and bad dreams. Siblings in the younger cohort were more likely to report feeling uncomfortable talking about their siblings diagnosis and less likely to report having someone to confide in or talk to about their siblings’ diagnosis compared to the older cohort.
A third and separate poster presented results from a Caregiver Benefit Scale for caregivers of children with epileptic encephalopathies and other chronic diseases. The results indicate caregivers of children with epileptic encephalopathies report lower benefits than caregivers from a community sample, but the benefit was similar to caregivers of children with Down syndrome and Muscular Dystrophy.
“These data suggest that the frequency and unpredictability of seizures in Dravet syndrome and other epileptic encephalopathies create a heavy burden that significantly impact the quality of life of the entire family unit,” said
Socioeconomic Impact of Dravet Syndrome in
A study of the socioeconomic burden of illness associated with Dravet syndrome within the German healthcare system found patient seizure burden was the major contributor to direct costs and incurred substantial other costs, such as specialist care or therapy for additional symptoms.
- 77% of patients experienced at least one seizure in the past month and 30% experienced status epilepticus in the previous 12 months.
- 89% of patients/caregivers have a severely disabled pass and 76% require significant to extreme categories of nursing care.
Durability and Safety of ZX008 in Belgian Dravet syndrome Cohort
Updated results from the Belgian, open-label prospective study of fenfluramine in patients with Dravet syndrome continue to demonstrate the long-term durability of effect with patients experiencing a median 87% reduction in convulsive seizures over the entire observation period (N=15, median 36 months; range 9 to 80 months) compared to the 3-month baseline period. Additionally, 67% of subjects experienced a ≥ 75% reduction in convulsive seizure frequency per month compared to baseline.
Fenfluramine was generally well-tolerated and demonstrated a safety profile consistent with the prior reports from the Belgian cohort. No patients exhibited cardiac valvulopathy or pulmonary hypertension at any time in the study. The most common adverse events were anorexia (n=11), sleepiness (n=9), fatigue (n=8), and mood changes (n=8).
“Combined with the data from our recently completed Phase 3 pivotal trials for ZX008 in Dravet syndrome, we are pleased with the durability of treatment effect that has been observed with fenfluramine in this cohort of patients who continue to experience clinically meaningful reductions in seizure frequency,” said
Dravet syndrome is a rare, severe and intractable form of epilepsy that begins in infancy with frequent and/or prolonged seizures.1 Studies have reported an incidence rate for Dravet syndrome of approximately 1 per 16,000 live births with onset occurring within the first year of life. Patients and caregivers managing these epilepsies – especially those who have been unable to control seizures with other treatments – experience daily, frequent and/or prolonged seizures that significantly impact their daily quality of life.2 Many caregivers must quit their jobs to care for their affected child, a commitment that results in substantial financial, physical, psychosocial and emotional burden.3
ZX008 is designated as an orphan drug as it pertains to Dravet syndrome and LGS in both the U.S. and Europe, and has received Breakthrough Therapy designation in the U.S. for the treatment of Dravet syndrome. ZX008, fenfluramine HCl oral solution, is an investigational compound that is being developed as a potential treatment for seizures in patients with Dravet syndrome and Lennox-Gastaut syndrome. It is currently not approved by any regulatory authorities to treat any condition.
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- Wu, E., et. al. (2015). Incidence of Dravet Syndrome in a US Population. Pediatrics 136(5): 1310-e1315. doi: 10.1542/peds.2015-1807.https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4621800/
- Aras LM, Isla J, Mingorance-Le Meur A. The European patient with Dravet syndrome: results from a parent-reported survey on antiepileptic drug use in the European population with Dravet syndrome. Epilepsy Behav. 2015 Mar;44:104-9.
Campbell, et.al. Epilepsy & Behavior 80 (2018) 152–156.
Source: Zogenix, Inc.