FDA Breakthrough Therapy Designation is intended to expedite the development and review of medicines aimed at treating a serious or life-threatening disease where there is preliminary clinical evidence that the investigational therapy may offer substantial improvement over existing therapies on at least one clinically significant endpoint.
FDA Breakthrough Therapy Designation for ZX008 is based on the results from Study 1, Zogenix’s first global Phase 3 trial of ZX008, which met the primary efficacy endpoint, as well as all prespecified key secondary efficacy endpoints.
Zogenix chief development officer Gail Farfel said: “We are very pleased that the FDA has granted Breakthrough Therapy Designation based on the efficacy and safety results from Study 1 reported in fall of 2017.
“We look forward to working closely with the FDA as we conclude our Phase 3 clinical program in Dravet syndrome, a rare and catastrophic form of childhood epilepsy.”
ZX008 is designated as an orphan drug in both the US and Europe for Dravet syndrome and Lennox-Gastaut syndrome, and has received Fast Track designation in the U.S. for the treatment of Dravet syndrome.