Zogenix announced new Phase 3 findings of ZX008 (low-dose fenfluramine HCl) for the treatment of Dravet syndrome at the AES Annual Meeting 2017.
Study 1 demonstrated that treatment with ZX008 0.8mg/kg/day led to 29 times higher odds of achieving a clinically meaningful (≥50%) reduction and 50 times higher odds of a substantial (≥75%) reduction in convulsive seizure frequency. About half of the patients (55%) treated with ZX008 were rated by parents and caregivers as very much improved or much improved in overall condition on the Clinical Global Impression (CGI-C) rating vs 10% of patients who received placebo (P=0.001). Also, 62.5% of patients treated with ZX008 were rated by investigators as very much improved or much improved in overall condition on the CGI-C vs 10% of the placebo group (P=0.001).
Initial findings, presented in September 2017, showed that ZX008 0.8mg/kg/day met its primary objective of superiority to placebo as adjunctive therapy in the treatment of Dravet syndrome in children and young adults based on change in the frequency of convulsive seizures between the 6-week baseline observation period and the 14-week treatment period (P<0.001). Compared to placebo, ZX008-treated patients experienced a 63.9% reduction in mean monthly convulsive seizures; the median percent reduction in monthly convulsive seizure frequency was 72.4% vs 17.4% in ZX008 and placebo groups, respectively.
“These new data demonstrate Dravet syndrome patients treated with ZX008 achieved a clinically meaningful reduction in seizure frequency. If approved, ZX008 could play an important role in changing the treatment paradigm for patients and their families whose lives have been greatly impacted by the lack of effective seizure control provided by current treatment options,” stated lead author LIeven Lagae, MD, phD, from University of Leuven, Belgium.
ZX008 has received Orphan Drug designation and Fast Track designation by the Food and Drug Administration (FDA) for the treatment of Dravet syndrome.