Rare Daily Staff

Rare disease drug developer Zogenix says it has reached an agreement with the U.S. Food and Drug Administration to resubmit its new drug application for Fintepla fenfluramine for the treatment of seizures associated with Dravet syndrome in the third quarter of this year.

Dravet syndrome is a rare genetic form of epilepsy caused by a mutation in the SCN1A gene. The severity of cognitive impairment is related to the frequency and duration of seizures. It occurs in about 1 in 300,000 people and currently there is no cure.

Zogenix met with the FDA at the end of May to discuss review the two issues the agency identified when it refused to accept the company’s first application in April. These issues included the absence of certain non-clinical studies to allow assessment of the chronic administration of fenfluramine, and the inclusion of an incorrect version of a clinical dataset in the submission.

The FDA agreed to Zogenix’s plan to resubmit the application without the inclusion of the new chronic toxicity studies. Zogenix also conducted and discussed with the FDA an analysis to explain the incorrect clinical dataset submitted in the original application, and the FDA is in agreement with the company’s plan for resubmission of the datasets in the NDA.

“We now have the clarity required to successfully resubmit our Fintepla NDA, which we anticipate will occur in the third quarter,” said Stephen Farr, president and CEO of Zogenix.

Separately, the FDA rescinded its Breakthrough Therapy designation for Fintepla for the treatment of seizures associated with Dravet syndrome because there are now two approved therapies for the disease, Epidiolex (cannabidiol), marketed ty Greenwich Biosciences in the United States, and Diacomit (stiripentol), produced by Biocodex.

The results from the Fintepla clinical program demonstrated that patients adding Fintepla to a stiripentol-based regimen experienced a 54.7 percent greater reduction in mean monthly convulsive seizure frequency compared to patients adding placebo. However, no such comparisons were possible with cannabidiol since it was an investigational product at the time the Fintepla pivotal trials were conducted and was therefore an excluded treatment.

Zogenix said it does not expect the rescission of Breakthrough Therapy designation to limit the potential for Priority Review status for the its resubmitted application.

Photo: Stephen Farr, president and CEO of Zogenix.

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