Currently Viewing:
Newsroom
Currently Reading
Low-Dose Fenfluramine Performs in Dravet Syndrome in Second Phase 3 Clinical Trial
August 01, 2018 – Kelly Davio
QR Code–Linked System Could Improve Adrenal Crisis Management in Addison Disease
July 25, 2018 – Alison Rodriguez
Study Finds Acetazolamide May Be Effective in Treating Glioblastoma
July 22, 2018 – Alison Rodriguez
Researchers Find Astrocytes to Be Central Driver Behind Huntington Disease Pathology
July 18, 2018 – Alison Rodriguez
Lomitapide Significantly Lowers LDL-C in Patients With HoFH, Study Finds
July 15, 2018 – Alison Rodriguez
DMD Trial Results Demonstrate Decrease in Creatine Kinase Levels
July 10, 2018 – Alison Rodriguez
FDA Approves of First Marijuana-Based Drug to Treat Rare Forms of Epilepsy
July 08, 2018 – Alison Rodriguez
Study Encourages Mixed Methods Research for Rare Disease Clinical Trials
July 02, 2018 – Alison Rodriguez
Challenges with Treating Merkel Cell Carcinoma and Other Rare Diseases
July 01, 2018 – Alison Rodriguez

Low-Dose Fenfluramine Performs in Dravet Syndrome in Second Phase 3 Clinical Trial

Kelly Davio
Fenfluramine, a drug that was previously sold as an appetite suppressant before it was withdrawn from the market in 1997 over concerns about its link to heart valve problems, may have a new application; low doses of fenfluramine hydrochloride have now proven successful in treating Dravet syndrome, a particularly severe form of epilepsy.
 
Fenfluramine, a drug that was previously sold as an appetite suppressant before it was withdrawn from the market in 1997 over concerns about its link to heart valve problems, may have a new application; low doses of fenfluramine hydrochloride have now proven successful in treating Dravet syndrome (DS), a particularly severe form of epilepsy.

DS, a severe, infantile-onset, highly treatment-resistant epileptic encephalopathy, is associated with an elevated risk of premature death and has few treatments available.

Zogenix, Inc., a pharmaceutical company developing a 0.5 mg/kg/day dose of fenfluramine under the name ZX008, this month revealed positive top-line results from its second phase 3 study of the investigational treatment for children and adults with DS. Like the first phase 3 trial, this study met its primary endpoint—demonstrating superiority to placebo—and both key secondary endpoints.

According to Zogenix, patients who took ZX008 had a 54.7% greater reduction in mean monthly convulsive seizures versus those who received placebo (P =.001), and the median reduction in monthly convulsive seizure frequency was 62.7% for those receiving the study drug versus 1.2% in those receiving the placebo. Patients receiving the study drug also showed improvements in the key secondary measures of clinically meaningful reductions (50% or more) in seizure frequency and longest seizure-free intervals.

Notably, the incidence of adverse events (AEs) was similar between the drug and placebo arms, and prospective cardiac safety monitoring throughout the study did not identify evidence of cardiac AEs in the patients.

These findings confirm those of the first phase 3 study, results of which were presented in 2017 at the American Epilepsy Society’s Annual Meeting; in the first study of 119 patients, the group who received ZX008 also experienced statistically significant, clinically meaningful reductions in seizure frequency compared with the placebo group, and no clinical or echocardiographic evidence of cardiac valvulopathy or pulmonary hypertension were observed.

“These impressive study results show the significant impact the addition of ZX008 made in reducing the burden of convulsive seizures for patients who are not adequately controlled using stiripentol, the standard of care for the treatment of Dravet syndrome in Europe,” said Professor Rima Nabbout, MD, PhD, principal investigator of the study, in a statement. “If approved, ZX008 has the potential to be a transformative treatment for [DS.]”

Zogenics reports that it is now preparing its regulatory applications for submission to US and European regulators by the end of the year.

 
Copyright AJMC 2006-2020 Clinical Care Targeted Communications Group, LLC. All Rights Reserved.
x
Welcome the the new and improved AJMC.com, the premier managed market network. Tell us about yourself so that we can serve you better.
Sign Up