Zogenix has announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for Fintepla (ZX008), its investigational compound for the treatment of epileptic seizures associated with Dravet syndrome.
Fintepla is a low-dose oral solution of fenfluramine hydrochloride. Patients take it with other treatments to reduce the frequency of epileptic seizures.
A prospective, post-hoc merged analysis was undertaken of Study 1. This randomized, double-blind, and placebo-controlled trial — a merge analysis of two identical studies, ZX008-1501 and ZX008-1502 — was designed to assess the efficacy and safety of Fintepla at a dose of 0.2 mg/kg/day and 0.8 mg/kg/day in 119 children and young adults with Dravet syndrome over a period of 14 weeks compared with a placebo.
Results revealed that, when compared to placebo, patients treated with the higher dose of Fintepla had much higher odds of achieving a clinically meaningful (more than 50%) or substantial (more than 75%) reduction in convulsive seizure frequency.
Results from Study 1504 were recently presented during the 72nd American Epilepsy Society (AES) Annual Meeting, and showed that after 14 weeks of treatment with Fintepla, patients achieved meaningful improvements in behavior, emotion, and cognitive regulation compared with those on placebo.
When used with other anti-epileptic drugs, such as Diacomit (stiripentol), Fintepla was highly tolerated and effective at reducing the frequency of seizures when used in combination with other anti-epileptic agents, compared with the placebo.
Data regarding 232 participants treated for up to 21 months participating in the ongoing Phase 3 open-label extension study (NCT02823145) has shown Fintepla is highly tolerable and safe over extended periods of time, with no patients developing cardiac or pulmonary complications associated with treatment. “Together with the efficacy data from the Phase 3 trials, fenfluramine [Fintepla] appears to have a positive benefit-risk profile in this patient population,” researchers stated.
“Our concurrent submissions to the FDA and EMA are the culmination of four years’ effort for Zogenix, our investigators, and the families who participated in the ZX008 clinical trial program,” Stephen J. Farr, president and chief executive officer of Zogenix, said in a press release. “We are honored to have partnered with such dedicated people to develop a potential new treatment for this rare and often catastrophic disease and look forward to working closely with the FDA and EMA during the review process.”
Zogenix is also investigating Fintepla in Lennox-Gastaut syndrome.