NDA for Low-Dose Fenfluramine Submitted for Treatment of Dravet Syndrome

Zogenix announced the submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA) for Fintepla (low-dose fenfluramine; ZX008) oral solution for the treatment of seizures associated with Dravet syndrome.

An amphetamine derivative, fenfluramine was initially developed as an appetite suppressant that is believed to work through serotonergic mechanisms. In the 1990s, the drug was withdrawn by the FDAdue to cardiovascular safety concerns, however it continues to be investigated for other therapeutic indications.

The NDA included data from two Phase 3 trials in Dravet syndrome and an interim analysis from an ongoing open-label extension study (Study 1503; N=232) of patients treated up to 21 months; the median duration of treatment was 256 days.

Results from Study 1503 showed a median 66.8% reduction in monthly convulsive seizure frequency over the entire open-label extension treatment period. Specifically, 64.4% of children and young adults achieved a >50% reduction in convulsive seizure frequency, and 41.2% achieved a >75% reduction. In addition, the rate of adverse events was similar to the Phase 3 placebo-controlled studies, with no signal of cardiovascular toxicity.

Fintepla was previously granted Orphan Drug, Fast Track, and Breakthrough Therapy designations by the FDA. The investigational product is also being evaluated in an ongoing Phase 3 trial for the treatment of Lennox-Gastaut syndrome.

For more information visit Zogenix.com.