Rare pediatric epilepsy new drug! Fintepla (Finteplamin) Treatment of Dravet Syndrome Phase 3 Phase III Clinical: Significant reduction in convulsive seizures!

!-- webeditor: page.title-- September 13, 2020 // -- Zogenix is a pharmaceutical company dedicated to the development of treatments for rare diseases.
recently, the company published positive top-line results from the third Phase 3 study (Study 3) on the treatment of Dravet syndrome-related epilepsy with fenfluramine oral solution.
study confirmed that Findepla had a substantial effect on the reduction of convulsive seizures in patients with this severe, rare, debilitating infant seizure in early Phase 3 trials (Studies 1 and 2).
also expanded the countries reviewed by Findepla, including Japan.
3 will be a key study for the company's plan to submit a new drug application (J-NDA) in Japan in 2021.
June, the FDA approved Fintepla oral solution CIVE for patients older than 2 years of age to treat epilepsy associated with Dravet syndrome.
the drug was approved through the FDA's priority review process.
, Fintepla is also under review by the European Medicines Agency (EMA).
March 2019, Zogenix and Nippon Shinyaku of Japan reached an exclusive distribution agreement for The commercialization of Findepla in Japan.
Zogenix will provide products to Japan's New Drug Co., Ltd. and will be responsible for completing Finetepla's global clinical development projects, including support for Zogenix's project to submit applications for new drugs for Dravet syndrome and Lennox-Gastaut syndrome in Japan.
Dravet syndrome is a rare childhood epilepsy characterized by frequent and severe drug-resistant seizures, associated hospitalization and medical emergencies, severe developmental and motor disorders, and an increased risk of sudden and unexpected death (SUDEP).
Fintepla is a low-dose fenfluramin for liquid preparations that reduces the frequency of seizures by regulating serotonin receptor and sigma-1 receptor activity (see reference: Fenfluramine designes NMDA receptor-mediatedd via its mixed activity at serotonin 5HT2A and type 1 sigma receptors, ).
data from two placebo-controlled Phase III clinical trials (Studies 1 and 2) showed that Fintepla significantly reduced the frequency of convulsive seizures compared to placebos in patients with other drugs that did not adequately control seizures.
addition to Dravet syndrome, Zogenix is also developing Findepla to treat seizures associated with Lennox-Gastaut syndrome (LGS).
Dravet syndrome and LGS are two rare and often catastrophic childhood seizures, characterized by early onset, diverse types of seizures, high frequency of seizures, severe damage to intelligence, and difficulty in treatment.
the United States, Fintepla was awarded breakthrough drug eligibility (BTD), orphan drug eligibility (ODD) for Dravet syndrome and LGS.
finfluolamine-molecular structure (Photo: Wikipedia.org) Study 3 is a multi-country, randomized, double-blind, placebo-controlled Phase 3 study that included 143 children and young people with Drafet syndrome who had seizures that could not be adequately controlled with existing anti-epileptic drugs.
the average baseline convulsion frequency was approximately 63 seizures per month in patients with a medium age of 9 years (range: 2-18 years).
After a six-week baseline observation period, patients were randomly divided into three treatment groups: Fintepla 0.7mg/kg/day (maximum daily dose of 26mg; n=49), 0.2mg/kg/day (n=46), placebo (n=48).
, Fintepla or placebo was added to each patient's current anti-epileptic drug treatment program.
patients were titrated to the target dose of Fintepla within 2 weeks and then continued for 12 weeks at that fixed dose.
results showed that the study achieved the main goal: patients in the Fintepla 0.7mg/kg/day group had an average monthly reduction in convulsive seizures by 64.8% compared to the placebo group.
the percentage of monthly convulsive seizures decreased to 73.7 percent in the Fintepla 0.7mg/kg/day group and 7.6 percent in the placebo group.
a key secondary goal of the study was to perform the same comparative analysis of low-dose Findepla (0.2mg/kg/day) with a placebo.
data showed that patients in the Fintepla 0.2mg/kg/day group had an average 49.9% reduction in convulsive seizures per month compared to the placebo group.
, these top-line data were highly consistent with the results of study 1, suggesting that Findepla had a dose-reactive relationship in the treatment of convulsive epilepsy with Dravet syndrome.
other key secondary objectives of the study were to compare Findepla 0.7 mg/kg/day and 0.2 mg/kg/day with a placebo in the following areas: (1) the proportion of patients with a reduction of more than 50% per month of convulsive seizures;
these results are shown in the table below, which also includes a 75% reduction in seizures in patients, which is a secondary efficacy indicator.
in the study, Fintepla was generally well-to-do, consistent with adverse events observed in Study 1 and Study 2, and consistent with The safety known to Findepla.
the risk of adverse events (TEAE) during treatment in the Fintepla treatment group was higher than in the placebo group: 91.7% of patients in the 0.7 mg/kg/day group (n=44) In the 0.2 mg/kg/day group, 91.3% of patients (n-42) had at least one TEAE, compared with 83.3% (n-40) in the placebo group.
rates of severe adverse events were similar in 3 groups, with 6.3% of patients in the 0.7 mg/kg/day group and 6.5 in the 0.2 mg/kg/day group Patients who experienced at least one severe TAAE in % (n-3) experienced 4.2% (n-2) in the placebo group, including one placebo patient who died as a result of SUDEP (sudden death from epilepsy).
, prospective heart safety monitoring showed no studies of valve heart disease or pulmonary hypertension in patients.
() !--/ewebeditor:page--!--ewebeditor:page-title"--the original source: Zogenix Announces Positive Top-Line Results from its Third Pivotal Phase 3 Clinical Trial (Study 3) of FINTE? in Dravet !--/ewebeditor:page.