Zogenix Announces FDA Acceptance for Filing of New Drug Application and Priority Review for FINTEPLA for the Treatment of Dravet Syndrome

Posted on November 25, 2019 by Medtech[y] Staff

Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for FINTEPLA (ZX008, fenfluramine oral solution) for the treatment of seizures associated with Dravet syndrome. The FDA granted Priority Review for the NDA for FINTEPLA, which provides for a six-month review from the date of receipt, and assigned a Prescription Drug User Fee Act (PDUFA) target action date of March 25, 2020.

“The FDA’s acceptance for filing of the NDA under Priority Review for our investigational product, FINTEPLA, represents a significant milestone in its development,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix. “We are excited to continue working with the FDA with the goal of bringing FINTEPLA to Dravet syndrome patients and their families in need as quickly as possible.”

Priority review is granted for investigational therapies that, if approved, may offer significant improvements in the treatment, prevention or diagnosis of a serious condition.

In its NDA filing acceptance letter, the FDA also stated that it is not currently planning to hold an advisory committee meeting to discuss the application for FINTEPLA.

The NDA is based on data from two positive pivotal Phase 3 trials in Dravet syndrome and an interim analysis from an ongoing open-label extension study, which included 232 patients treated for up to 21 months. Zogenix is also investigating FINTEPLA in Lennox-Gastaut syndrome, another rare, severe childhood-onset epileptic syndrome, for which a Phase 3 trial is ongoing, with top-line data results anticipated 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 has two late-stage development programs underway: FINTEPLA® (fenfluramine) for the treatment of seizures associated with Dravet and Lennox-Gastaut syndromes, two rare and often-catastrophic childhood-onset epilepsies. Additionally, Zogenix is in clinical development with MT1621, a novel, investigational substrate enhancement therapy for the treatment of a rare genetic disorder called TK2 deficiency. The U.S. Food & Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for FINTEPLA for the treatment of seizures associated with Dravet syndrome. The FDA granted Priority Review for the NDA for FINTEPLA. The marketing authorization application for FINTEPLA for Dravet syndrome is under review by the European Medicines Agency. Zogenix expects top-line data results from its Phase 3 study of FINTEPLA in Lennox-Gastaut syndrome in the first quarter of 2020. FINTEPLA is also in development in Japan for Dravet and Lennox-Gastaut syndromes.

Forward-Looking Statement 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 timing and results of any decision regarding the NDA from the FDA; and the potential timing of top-line data for the Phase 3 study of FINTEPLA in Lennox-Gastaut syndrome (Study 1601). These statements are based on Zogenix’s current beliefs and expectations. 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 release due to the risks and uncertainties inherent in Zogenix’s business, including, without limitation: the potential for the FDA to delay the PDUFA target action date due to the FDA's internal resource constraints or other reasons; the FDA may later determine to hold an advisory committee meeting and risks associated therewith; the FDA may disagree that the existing safety and efficacy data is sufficient to approve the NDA; the FDA may require Zogenix to conduct the additional chronic toxicity studies noted in the Refusal to File letter, dated April 3, 2019, or other studies or information in connection with its review of the NDA; the timing of the data from Study 1601 of FINTEPLA in patients suffering from Lennox-Gastaut syndrome could be delayed; additional data from Zogenix’s ongoing studies may contradict or undermine the data submitted in the NDA for FINTEPLA; the uncertainties associated with the clinical development and regulatory approval of product candidates such as FINTEPLA and MT1621; unexpected adverse side effects or inadequate therapeutic efficacy of FINTEPLA that could limit approval and/or commercialization, or that could result in recalls or product liability claims; and other risks described in Zogenix’s prior press releases as well as in public periodic filings with the U.S. Securities & Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and Zogenix 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.