The active IND now allows the Company to initiate its planned Phase 3 program for ZX008.
Zogenix president and CEO Stephen Farr said: "We are pleased to have reached agreement with the FDA to proceed into Phase 3 clinical development, and are now focused on initiating the U.S.-based pivotal clinical trial for ZX008 prior to year-end, which would position us well to generate top-line results in 2016.
"This is a significant milestone for Zogenix, as we believe ZX008 has the potential to provide Dravet patients and their families with an important treatment option for this catastrophic form of intractable epilepsy that begins in infancy."
The Phase 3 program for ZX008 will consist of two randomized, double-blind placebo-controlled studies that will include two dose levels of ZX008 (0.2 mg/kg/day and 0.8 mg/kg/day, up to a maximum daily dose of 30 mg), as well as placebo. Zogenix intends to enroll 105 subjects in each of the two studies, with 35 patients in each treatment arm.
One study will be conducted primarily in the U.S. and Canada, and the other will be a multi-national study, conducted primarily in Europe.
The primary endpoint will be the change in frequency of convulsive seizures as compared to placebo. The key secondary endpoints include 40% and 50% responder analyses and convulsive seizure-free interval.