The FDA has granted Zogenix Inc. breakthrough therapy designation for low-dose fenfluramine use to treat seizures related to Dravet syndrome, according to a press release issued by the company.
Dravet syndrome is a severe form of epilepsy caused by genetic dysfunction within the brain. Most people with the condition experience onset within the first year of life. Seizures are often accompanied by fever. This syndrome is considered an intractable or uncontrollable condition.
ZX008 has reached both primary and secondary efficacy endpoints for the treatment of seizures caused by Dravet syndrome in a phase 3 trial and has received fast track designation within the United States. The drug also is designated as an orphan drug in Europe.
“We are very pleased that the FDA has granted breakthrough therapy designation based on the efficacy and safety results from Study 1 reported in the fall of 2017,” Gail M. Farfel, PhD, chief development officer of Zogenix, said in the release. “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 can also be used for the treatment of Lennox-Gastaut syndrome, according to the release This form of epilepsy is also severe and begins in childhood. Those who have this condition are likely to also have intellectual disability.
The FDA grants breakthrough therapy designation to manufacturers who are developing medications intended for use in serious and life-threatening diseases and conditions. These medications require preliminary clinical evidence that its effects provide significant improvement in at least one clinically significant endpoint when compared to other available therapies.