Zogenix gains Breakthrough status, but GW Pharma still ahead in Dravet syndrome

The FDA has granted Zogenix a Breakthrough Therapy Designation for its pipeline product, ZX008 for the treatment of seizures associated with Dravet syndrome.

The news came yesterday amid a run on shares around the world, but Zogenix managed to buck the trend, rising more than 8% thanks to the significance of the FDA decision.

The Breakthrough Therapy Designation (BTD) is good news for Zogenix, but rival GW Pharma is still in the lead in the field, and is expected to gain US and European approval this year.

The breakthrough status is based on results from Study 1, Zogenix’s first global phase 3 trial of ZX008, which met the primary efficacy endpoint, as well as all pre-specified key secondary efficacy endpoints.

Dravet syndrome is a rare form of intractable epilepsy, for which there are currently no effective treatments.

“We are very pleased that the FDA has granted Breakthrough Therapy Designation based on the efficacy and safety results from Study 1 reported in fall of 2017,” said Gail M. Farfel, chief development officer of Zogenix. “We look forward to working closely with the FDA as we conclude our phase 3 clinical programme in Dravet syndrome, a rare and catastrophic form of childhood epilepsy.”

ZX008 is designated as an orphan drug in both the US and Europe for Dravet syndrome and Lennox-Gastaut syndrome, and has received Fast Track designation in the US for the treatment of Dravet syndrome.

Rivals GW Pharma unveiled phase 3 for its own candidate in December. Its Epidiolex (cannabidiol or CBD) now has late-stage data for Dravet syndrome and Lennox-Gastaut syndrome (LGS).

After establishing its BTD system predominantly in oncology, the FDA is now shifting its emphasis to the CNS field. Last month it picked out Roche’s Autism Spectrum Disorder candidate balovaptan for the fast-track status.