New Data Could Boost Potential Approval of Zogenix's Epilepsy Drug

Shares of Zogenix are climbing this morning after the California-based company posted new positive data for its investigational drug Fintepla in Dravet syndrome, a rare type of epilepsy. The new data could boost the company’s chances of securing regulatory approval.

Poster presentations unveiled at the recent Childhood Neurology Society (CNS) Congress showed that Fintepla (fenfluramine) provides long-term and clinically meaningful convulsive seizure reduction in 75.5% of Dravet syndrome patients under the age of 6 years who were treated in a Phase III trial. This compared to a median decrease of 60.1% in the older, over-6 years of age group and a median decrease of 63.6% in the overall study population, ages 2-18 years, the company said. Fintepla was generally well-tolerated in this study and no case of valvular heart disease or pulmonary arterial hypertension was observed in any patient at any time.

Additionally, a post-hoc analysis of previous Phase III studies showed a “profound reduction” in high-risk tonic-clonic (grand-mal) seizures patients, the company said. The analysis showed that the median baseline monthly frequency of generalized tonic-clonic seizures ranged from 8 to 12.3 per month. Those seizures decreased by 80%, 64%, and 48% in the Fintepla groups, respectively, compared to 10% in the placebo group, Zogenix said.

Now Hiring  Currently hiring sales representatives, clinical researchers, engineers,  science r&d professionals, and other disciplines. Browse Jobs

For Zogenix, the new positive data regarding Fintepla is important particularly after the U.S. Food and Drug Administration rejected the company’s New Drug Application earlier this year. In April the FDA issued a Refusal to File letter for the drug. The regulatory agency said the NDA was not “sufficiently complete to permit a substantive review.” The rejection raised two concerns. The first regarded non-clinical studies that were not submitted to allow assessment of the chronic administration of fenfluramine, the active ingredient in Fintepla. The second reason cited was that the application contained an “incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA.”

The company resubmitted its NDA filing in September.

Regarding the new data, Zogenix Chief Medical Officer Bradley Galer said the clinical data continues to “demonstrate the significant clinical impact Fintepla has shown in studies of Dravet syndrome patients.” The results, Galer said, have positively impacted the vulnerable pediatric patients, as well as those suffering from grand-mal seizures, which are a “recognized risk factor for sudden unexplained death in epilepsy.”

Dravet syndrome, also called Severe Myoclonic Epilepsy of Infancy, is typically caused by loss-of-function mutations in the SCN1A gene. The genetic disorder is characterized by uncontrolled seizures, ataxia, significant developmental delays and an increased risk of early mortality due to sudden unexpected death in epilepsy.

Fintepla is also being developed as a potential treatment for Lennox-Gastaut syndrome, another rare epilepsy. Zogenix anticipates top-line data for Fintepla in Lennox-Gastaut syndrome in the first quarter of 2020.

Other data presented at the conference include the results of a Phase 1 study to assess the potential drug-drug interaction of fenfluramine and cannabidiol. The results of the study showed that the effects of CBD on fenfluramine are unlikely to require dose adjustments when the drugs are co-administered.

Back to news