RARE Daily

FDA Notifies Zogenix of Inadequacies in Dravet Syndrome Drug Application

April 9, 2019

The U.S. Food and Drug Administration notified Zogenix that its application for the approval of Fintepla for the treatment of seizures associated with Dravet syndrome could not be reviewed because it lacks necessary elements.

Upon its preliminary review, the FDA determined that the application Zogenix submitted on February 5 was not sufficient because certain non-clinical studies were not submitted to allow assessment of the chronic administration of Fintepla. The application also contained an incorrect version of a clinical dataset, which prevented the completion of the review process.

The agency has not requested or recommended additional clinical efficacy or safety studies. The setback will further delay Zogenix from being able to compete against GW Pharmaceuticals, which in November won FDA approval for Epidiolex for the treatment of seizures associated with both Dravet syndrome and Lennox-Gastaut syndrome.

Zogenix said it will seek immediate guidance, including a meeting with the FDA, to clarify and respond to the issues identified in the FDA’s letter.

“We remain highly confident in Fintepla’s clinical profile demonstrated in the phase 3 program in Dravet syndrome and are committed to advancing the product candidate as a potential new treatment option for this and other rare and often catastrophic epileptic encephalopathies,” said Stephen Farr, president and CEO of Zogenix. “We are fully committed to working with the FDA as quickly as possible to address the open issues and clarify the path to successfully re-filing our application.”

The company said its application for approval to market Fintepla for the treatment of seizures associated with Dravet syndrome was previously accepted for review by the European Medicines Agency. The company anticipates an approvability decision could be reached by the EMA in the first quarter of 2020.   

Dravet syndrome is a rare form of treatment-resistant epilepsy that begins in infancy and is associated with frequent, severe, and potentially life-threatening seizures, developmental delay, cognitive impairment, and an elevated risk of sudden unexplained death in epilepsy. The disease also has an impact on the entire family, often resulting in substantial financial, physical, psychosocial and emotional burdens. 

Fintepla is a low-dose form of Fenfluramine, a drug that was formerly sold as an appetite suppressant to treat obesity. It was withdrawn from the market due to safety concerns.

Zogenix is also investigating Fintepla in Lennox-Gastaut syndrome, another rare childhood-onset epilepsy, for which a Phase 3 trial is ongoing.

Photo: Stephen Farr, president and CEO of Zogenix

Author: Rare Daily Staff

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