Zogenix has won the FDA’s breakthrough therapy designation following some impressive Phase III data announced last fall, gaining an inside track at the agency for its closely watched Dravet syndrome drug ZX008.
A bit of a Wall Street darling at the moment, Zogenix $ZGNX has watched its stock soar over 175% since it released data on the first of two Phase III studies for this drug. The company reported ZX008, designed to tackle a severe form of childhood epilepsy, triggered a dramatic drop in the rate of seizures experienced by patients with Dravet syndrome. At the high end, the dose cut the monthly convulsive seizure frequency by 72.4% compared to a 17.4% drop in placebo patients.
“Not only did the clinical trial meet its primary endpoint with a high degree of statistical significance – the magnitude of clinical benefit was better than we expected, and in the ballpark of prior compassionate use studies that conveyed an unprecedented level of benefit,” wrote Leerink’s Paul Matteis in an investor’s note at the time of the announcement.
This new FDA designation will speed development and review of the drug, likely setting Zogenix up to compete with epilepsy contender GW Pharmaceuticals $GWPH. GW recently reported its drug Epidiolex showed notable effect against Dravet syndrome, in which it triggered a 39% reduction in convulsive seizures compared to a 13% placebo group in late-stage trials.
“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 Farfel, chief development officer at Zogenix, in a statement. “We look forward to working closely with the FDA as we conclude our Phase 3 clinical program in Dravet syndrome, a rare and catastrophic form of childhood epilepsy.”
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