FDA Grants Zogenix Breakthrough Therapy Designation for Experimental Dravet Syndrome Therapy

Rare Daily Staff

The U.S. Food and Drug Administration awarded Zogenix Breakthrough Therapy Designation for its investigational product, ZX008 (low-dose fenfluramine), for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy that begins in infancy and has life threatening consequences.

Children with Dravet syndrome experience severe, long-lasting, fever-related seizures in the first year of life, with other seizure types typically starting over the next several years. The seizures do not respond to standard anti-epileptic medications and often times result in cognitive and developmental impairment, and can be fatal. Children today are treated with available anticonvulsants, but outcomes remain poor as there are no effective, long-term treatments.

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 experimental therapy may offer substantial improvement over existing therapies on at least one clinically significant endpoint. 

The FDA based its Breakthrough Therapy designation on the results from Zogenix’s first global phase 3 trial of ZX008, which met the primary efficacy endpoint, as well as all pre-specified key secondary efficacy endpoints.

ZX008 is designated as an orphan drug in both the United States and Europe for Dravet syndrome and Lennox-Gastaut syndrome, and has received Fast Track designation in the United States for the treatment of Dravet syndrome.

February 6, 2018