ZX008 is an investigational, low-dose fenfluramine liquid solution for the treatment of Dravet syndrome, a rare form of epilepsy. Dravet syndrome usually starts in the first year of life and affects approximately 1 in 16,000 children born in the U.S.
Symptoms include frequent and prolonged seizures with other related complications such as growth, speech, movement, and balance issues that are usually noticed after the second or third year of life. Current antiepileptic drugs (AEDs) are usually not effective for these seizures. People with Dravet syndrome face mortality rates of 15 to 20 percent.
The active ingredient of ZX008, fenfluramine, was originally developed and approved as an appetite suppressant to treat obesity. In 1997, fenfluramine was taken off the market due to the risk of heart valve damage in people with heart-related conditions.
In previous studies, the drug showed anticonvulsive effects, which led the Belgium government to authorize its study in a small group of people diagnosed with Dravet syndrome.
ZX008 is currently being evaluated in several Phase 3 studies.
One of these, the double-blind, parallel group, placebo-controlled study (NCT02682927) aims to assess the effectiveness, safety, and pharmacokinetics (drug properties profile) of ZX008 as an add-on therapy. Participants are randomized to either receive ZX008 at adjusted doses of 0.2 mg per kg body weight a day or 0.8 mg per kg body weight a day (maximum of 30 mg per day) or a matching placebo over a 14-day treatment period. Participants will then be able to take part in open-label, extension study (NCT02823145) to assess the long-term effects of the treatment.
Another study (NCT02826863) to evaluate the effectiveness, safety, and pharmacokinetics of ZX008 is also recruiting participants. In this study participants will receive one of two doses of ZX008 per day or a placebo.
Finally, a double-blind, randomized, two-arm pivotal Phase 3 study (NCT02926898) is assessing the efficacy, safety, and pharmacokinetics of ZX008 in addition to stiripentol in children and young adults with Dravet syndrome. In February 2017, Zogenix announced the start of the safety and efficacy portion of a second double-blind, randomized, two-arm pivotal Phase 3 trial of ZX008 as an add-on therapy to a drug regimen that includes stiripentol, valproate, and clobazam.
Results from these Phase 3 studies are expected in 2017.
ZX008 has received orphan drug status in both the U.S. and Europe, as well as fast track designation in the U.S. for the treatment of Dravet syndrome.
The drug has a U.S. patent, titled “Method for the treatment of Dravet syndrome.”
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