Delisted "old drug" gets new FDA approval for breakthrough therapy to treat rare childhood epilepsy

Dravet syndrome is a rare childhood seizure characterized by frequent and severe refractive seizures, associated hospitalizations and medical emergencies, significant developmental and motor disorders, and an increased risk of unexpected sudden deathThe Fintepla, developed by Zogenix, is a liquid form of low-dose fenfluraminIt can reduce the frequency of seizures by regulating the serotonin mechanism and sigma-1 receptor activityIt has been granted orphan drugs in the United States and the European Union, and has been awarded breakthrough therapies by the FDAFDA approval is based on data from two randomized, double-blind, placebo-controlled Phase 3 clinical trials published in The Lancet and JAMA NeurologyThe safety data comes from an open-label extended trial in which many patients receive Fintepla treatment for up to three yearsIn patients who failed to adequately control seizures with one or more anti-epileptic drugs, Fintepla significantly reduced the frequency of seizures per month compared to placebolong-term open label extension trial data released in April this year showed that more than 37 percent of patients had more than 75 percent fewer seizures when the median treatment period reached 445 daysIn addition, long-term safety studies did not detect primary pulmonary hypertension and heart valve disease in patientsDrStephen JFarr, President and CEO ofZogenix, said, "FDA approval of Fintepla is an important milestone and we are proud to celebrate with patients and families of Dravet syndromeFenfluramin has different pharmacologies than all other anticonvulsantsWe would like to thank our patients, their families and all those who have supported Fintepla's rigorous research and development program"