The Lancet Publishes Phase 3 Trial Results for Zogenix Investigational Drug FINTEPLA® in Dravet Syndrome
FINTEPLA as adjunctive therapy showed a significant, dose-dependent reduction in convulsive seizure frequency in Dravet syndrome patients versus placebo
“The results from this study are tremendously encouraging in reducing the magnitude and duration of seizures in our patients with Dravet syndrome,” said
Study 1 was an international, double blind, placebo-controlled Phase 3 study of 119 Dravet syndrome patients ages 2-18 years (mean age 9 years) treated at sites in the U.S.,
The primary endpoint was the change in mean monthly frequency of convulsive seizures during the treatment period compared with baseline in the 0.7 mg/kg/day group versus placebo. Results showed that patients taking FINTEPLA at 0.7 mg/kg/day achieved a 62.3% greater reduction in mean monthly convulsive seizure frequency compared to placebo (95% CI -47.7 to -72.8, p<0.0001). The same analysis for the group treated at 0.2 mg/kg/day versus placebo was a key secondary endpoint, with results showing a 32.4% greater reduction in mean monthly convulsive seizure frequency compared to placebo (95% CI -6.2 to -51.3, p=0.0209).
During the treatment period, the median reduction in seizure frequency was 74.9% in the 0.7 mg/kg/day group (from median 20.7 per 28 days to 4.7 per 28 days), 42.3% in the 0.2 mg/kg/day group (from median 17.5 per 28 days to 12.6 per 28 days), and 19.2% in the placebo group (from median 27.3 per 28 days to 22.0 per 28 days).
In addition to the seizure frequency data described above, more patients treated with FINTEPLA during the study achieved a clinically meaningful (≥ 50%) reduction in convulsive seizure frequency compared to placebo: 27 (68%) of 40 patients in the 0.7 mg/kg/day group (p<0.0001) and 15 (38%) of 39 patients in the 0.2 mg/kg/day group (p=0.0091), compared with five (12%) of 40 patients in the placebo group. FINTEPLA also provided significantly longer periods of seizure freedom to patients in the study: the median longest seizure-free intervals were 25 days in the 0.7 mg/kg/day group (p=0.0001) and 15 days in the 0.2 mg/kg/day group (p=0.0352), compared to 9.5 days in the placebo group.
The most common adverse events (occurring in at least 10% of patients, and more frequently in the FINTEPLA groups), were decreased appetite, diarrhea, fatigue, lethargy, somnolence and decreased weight. Echocardiographic examinations revealed normal valve function and morphology in all patients during the trial and no signs of pulmonary arterial hypertension.
“We are proud that the prestigious journal, The Lancet, has published these important results for the international medical community and very much appreciate the investigators, patients and families who made this study possible,” said
*Study 1 data results were previously presented by
Zogenix is a global pharmaceutical company committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases. The company has two late-stage development programs underway: FINTEPLA® (ZX008, fenfluramine oral solution) for the treatment of seizures associated with Dravet and Lennox-Gastaut syndromes, two rare and often-catastrophic childhood-onset epilepsies, and MT1621, a novel substrate enhancement therapy for the treatment of a rare genetic disorder called TK2 deficiency. Applications for Zogenix’s investigational drug, FINTEPLA, for Dravet syndrome are under review by the
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Source: Zogenix, Inc