Drug for Dravet syndrome may offer hope
Dravet syndrome is a severe childhood epilepsy that typically begins in the first year of infancy. The prevalence of this rare disorder ranges from 1:20,000 to 1:40,000 births and is characterised by intractable seizures.
“We are extremely pleased to have advanced the ZX008 programme to the start of the Phase 3 trial. It is an important step toward making this potentially breakthrough treatment available to Dravet patients and their families,” said Gail M. Farfel, Ph.D., Chief Development Officer of Zogenix.
The US-based Phase 3 trial will include two randomised, placebo-controlled studies of two dose levels of the drug as well as a placebo. Zogenix intends to enrol 105 subjects in each study.
In addition, Zogenix will conduct a multi-national study primarily in Europe. This study is expected to begin in the first quarter of 2016.
It is hoped that both the US and European studies will demonstrate a reduction in frequency of convulsive seizures in subjects compared to the placebo.
“We are eager to begin this clinical trial as a potential new treatment option for children with Dravet syndrome,” said Dr. Joseph Sullivan, Associate Professor of Neurology & Paediatrics at University of California, San Francisco (UCSF), Director of the UCSF Paediatric Epilepsy Centre and Principal Investigator of the North American ZX008 trial.
“Effective treatment options for this catastrophic epilepsy syndrome are quite limited. The data generated so far for ZX008 have been encouraging. We look forward to seeing results from the Phase 3 clinical trial.”