Zogenix Receives Refusal to File Letter from U.S. Food and Drug Administration for FINTEPLA® New Drug Application

EMERYVILLE, Calif., April 08, 2019 (GLOBE NEWSWIRE) -- Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for FINTEPLA^® (ZX008, fenfluramine hydrochloride) for the treatment of seizures associated with Dravet syndrome.

Upon its preliminary review, the FDA determined that the NDA, submitted on February 5, 2019, was not sufficiently complete to permit a substantive review.  In the letter, the FDA cited two reasons for the RTF decision: first, certain non-clinical studies were not submitted to allow assessment of the chronic administration of fenfluramine; and, second, the application contained an incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA. The FDA has not requested or recommended additional clinical efficacy or safety studies.

The Company will seek immediate guidance, including a Type A meeting with the FDA, to clarify and respond to the issues identified in the RTF 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 J. Farr, Ph.D., 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.”

Zogenix’s Marketing Authorization Application (MAA) for FINTEPLA for the treatment of seizures associated with Dravet syndrome was previously accepted for review by the European Medicines Agency (EMA), and the Company anticipates an approvability decision could be reached by the EMA in the first quarter of 2020. 

About Zogenix

Zogenix is a global pharmaceutical company committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases. The company’s lead candidate, FINTEPLA® (ZX008, fenfluramine) has been accepted for review by the European Medicines Agency and is in development in Japan. For more information, visit .

Forward Looking Statements

Zogenix cautions you that statements included in this press release that are not a description of historical facts are forward-looking statements.  Words such as “believes,” “anticipates,” “plans,” “expects,” “indicates,” “will,” “intends,” “potential,” “suggests,” “assuming,” “designed,” and similar expressions are intended to identify forward-looking statements.  These statements include: the Company’s plans to seek immediate guidance from, and a Type A meeting with, the FDA and to address the issues identified in the RTF letter and re-file the NDA; and a potential approvability decision by the EMA on the MAA in the first quarter of 2020.  The inclusion of forward-looking statements should not be regarded as a representation by Zogenix that any of its plans will be achieved.  Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Zogenix’s business, including, without limitation:  the timing, occurrence and outcome of the planned Type A meeting with the FDA to discuss the RTF letter; Zogenix’s ability to address the identified issues and successfully meet the FDA’s requirements for resubmission of the NDA and the timing thereof; the FDA may require additional non-clinical or clinical studies and the potential for the FDA to impose other requirements to be completed before or after approval of the NDA; the FDA or EMA may disagree that the existing safety and efficacy data is sufficient to allow an NDA or MAA, respectively, approval; Zogenix’s reliance on third parties for the preparation of clinical study reports and data analyses; the FDA and/or the EMA may not agree with Zogenix’s interpretation of the results of the clinical trials of FINTEPLA; additional data from Zogenix’s ongoing studies may contradict or undermine the data submitted in the NDA and MAA for FINTEPLA; the uncertainties associated with the clinical development and regulatory approval of product candidates such as FINTEPLA; potential delays by the EMA on an approvability decision, including as a result of the RTF letter; unexpected adverse side effects or inadequate therapeutic efficacy of FINTEPLA that could limit approval and/or commercialization; and other risks described in Zogenix’s prior press releases and filings with the U.S. Securities and Exchange Commission.  You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the Company undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.  All forward-looking statements are qualified in their entirety by this cautionary statement.  This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.

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