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LOS ANGELES -- Patients with Dravet syndrome may find a reduction in seizures with ZX008 (fenfluramine HCl oral solution) after failing treatment with stiripentol, a drug currently only available in the U.S. via a compassionate-use request. Stiripentol is approved for Dravet syndrome in Canada, Europe, Australia, and Japan.

Post-hoc analysis of a phase III trial of ZX008 showed a 60.8% reduction over placebo in mean monthly convulsive seizures with a 0.8 mg/kg/day dose (P=0.002), reported Elaine C. Wirrell, MD, of the Mayo Clinic in Rochester, Minn. At a lower 0.2-mg/kg/day dose there was a nonsignificant 35.1% reduction over placebo.

Among the 22 patients randomized to receive the 0.8 mg/kg/day dose, 86.4% saw a 25% reduction in major motor seizures over 28 days (OR 23, P=0.001), 72.7% patients saw a 50% reduction (OR 24.7, P=0.006), and 50% saw a 75% reduction (OR 15.8, P=0.036).

There were more treatment-emergent adverse events reported in those receiving the higher dose, the most common of which being decreased appetite -- unsurprising since fenfluramine was once used in Fen-Phen, the weight-loss combination drug that was pulled from the market by the FDA in the 1990s.

In a cardiovascular safety analysis, there were no vases of cardiac valvulopathy reported throughout the 2-week titration or 12-week maintenance phases of the study.

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