Low-Dose Fenfluramine Successful in Treating Dravet Syndrome in Phase 3 Trial

Fenfluramine, a drug that was previously sold as an appetite suppressant before it was withdrawn from the market in 1997 over concerns about its link to heart valve problems, may have a new application; low doses of fenfluramine hydrochloride have now proven successful in treating Dravet syndrome (DS), a particularly severe form of epilepsy.

DS, a severe, infantile-onset, highly treatment-resistant epileptic encephalopathy, is associated with an elevated risk of premature death and has few treatments available.

Zogenix, Inc., a pharmaceutical company developing a 0.5 mg/kg/day dose of fenfluramine under the name ZX008, this month revealed positive top-line results from its second phase 3 study of the investigational treatment for children and adults with DS. Like the first phase 3 trial, this study met its primary endpoint—demonstrating superiority to placebo—and both key secondary endpoints.

According to Zogenix, patients who took ZX008 had a 54.7% greater reduction in mean monthly convulsive seizures versus those who received placebo (P =.001), and the median reduction in monthly convulsive seizure frequency was 62.7% for those receiving the study drug versus 1.2% in those receiving the placebo. Patients receiving the study drug also showed improvements in the key secondary measures of clinically meaningful reductions (50% or more) in seizure frequency and longest seizure-free intervals.

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