Zogenix has confirmed the efficacy of its experimental epilepsy drug in a second, late-stage clinical trial, paving the way for a marketing submission by the end of the year. The small biotech company, based in Emeryville, Calif., said its lead drug, ZX008, reduced by more than half the average monthly convulsive seizures compared with a placebo in children and teenagers with Dravet syndrome, a rare and severe type of epilepsy.
Dravet syndrome is a very rare type of epilepsy that causes frequent and severe seizures, often beginning in the first year of life. While there is currently no definitive cure for Dravet syndrome, there are treatments available to reduce the number of seizures that can help make the condition more manageable.
Cannabidiol is an effective and generally well-tolerated add-on treatment for drop seizures in patients with Lennox-Gastaut syndrome, according to research published in The Lancet. “Patients with Lennox-Gastaut syndrome, a rare, severe form of epileptic encephalopathy, are frequently treatment resistant to available medications,” Elizabeth A. Thiele, MD, from the pediatric epilepsy program at Massachusetts General Hospital, and colleagues wrote. “No controlled studies have investigated the use of cannabidiol for patients with seizures associated with Lennox-Gastaut syndrome.”
A drug that has previously been used as a weight-loss agent dramatically reduces seizures in young patients with Dravet syndrome (DS), new research shows. A new phase 3 study showed that compared with patients taking placebo, those taking low-dose fenfluramine (ZX008, Zogenix) experienced a statistically significant reduction in convulsive seizure frequency. “The study showed a really significant and clinically meaningful decrease in seizure frequency. In about 70% of patients taking fenfluramine, the number of seizures per month went from about 40 to one or two per month,” lead author Lieven Lagae, MD, PhD, professor, University of Leuven, Belgium, head of the Pediatric Neurology Department, and director of the Childhood Epilepsy Program at the University of Leuv...
Global survey of 584 caregivers of children, young adults and adults with Dravet illustrates the detrimental effect of debilitating, frequent seizures on quality of life and co-morbidities: Results suggest a need for more effective antiepileptic treatments. The Dravet Syndrome European Federation and Zogenix, Inc. (NASDAQ:ZGNX), a pharmaceutical company developing therapies for the treatment of rare central nervous system (CNS) disorders, announced today the publication of the initial results from a landmark survey into the clinical, social and economic consequences of Dravet syndrome on children, young adults and their families in Developmental Medicine & Child Neurology.
The U.S. Food and Drug Administration has granted Orphan Drug Designation to EPX-100 and EPX-200 for the treatment of patients with Dravet syndrome. Dravet Syndrome qualifies as a rare pediatric disease under Section 529 of Food, Drug, and Cosmetic Act. The rare, catastrophic, lifelong form of epilepsy begins in the first year of life with frequent or prolonged seizures. Intellectual disability, behavioral abnormalities, gait and motor dysfunction, and increased mortality are commonly observed as the disease progresses. Patients with Dravet Syndrome also suffer with life-threatening seizures that cannot be adequately controlled by available medications, and face a 15-20 percent mortality rate due to SUDEP (Sudden Unexplained Death in Epilepsy), seizure-related accidents such as drowning, o...
What is sudden unexpected death in epilepsy (SUDEP)? Currently, the accepted definition of SUDEP is the sudden unexpected witnessed or unwitnessed, non-traumatic and non-drowning death in people with epilepsy, with or without evidence of a seizure. It doesn’t necessarily have to be in the known context of a seizure, excluding documented status epilepticus, where people have seizure after seizure. Typically, what happens is that someone who’s known to have epilepsy is found to have died without any obvious explanation as to why. Even a post-mortem examination doesn’t reveal a structural or toxicological cause of death. It may or may not be known whether a seizure had occurred and no other cause of death has been identified.