The designation follows a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency (EMA).
Stephen J. Farr, PhD, president and CEO of Zogenix, called the decision “an important component in our strategy to evaluate potential expanded uses” of ZX008 in other rare and severe forms of epilepsy.
Farr said in a press release that his company presented “compelling preliminary efficacy data” from an ongoing, open-label Phase 2 study [NCT02655198] for ZX008 in LGS at the American Epilepsy Society’s annual meeting last December. “Based on these promising results, we intend to move forward with a Phase 3 clinical study for ZX008 in LGS in the second half of 2017,” he said.
Zogenix, a pharmaceutical company based in Emeryville, California, will now receive protocol assistance, access to the European Union’s centralized authorization procedure with a single application, market exclusivity for 10 years after approval, fee reductions on regulatory activities and other EU incentives.
The EMA awards orphan drug designation to investigational treatments for rare disorders that cannot be treated. The EU defines rare diseases as those affecting no more than five in 10,000 Europeans.
LGS is a refractory, debilitating childhood-onset epilepsy accounting for 2 to 5 percent of childhood epilepsy, according to the Epilepsy Foundation. Seizures caused by this type of epilepsy are hard to control and children will need lifelong treatment. Intellectual and behavioral problems add to the complexity of this syndrome as well as the difficulties in managing these patients’ lives.
ZX008 is a low-dose fenfluramine formulated for pediatric use. It is now being evaluated to reduce seizures in patients with Dravet syndrome, another type of severe childhood epilepsy. The ZX008 Phase 3 program in Dravet syndrome includes four Phase 3 clinical studies being conducted in the United States and elsewhere (NCT02823145, NCT02926898, NCT02682927, NCT02826863).
In January 2016, the U.S. Food and Drug Administration granted ZX008 fast-track designation for treating Dravet syndrome.