Zogenix Submits Type II Variation Application to the European Medicines Agency (EMA)
Zogenix Submits Type II Variation Application to the European Medicines Agency (EMA) to expand the use of FINTEPLA® (Fenfluramine) for the Treatment of Seizures Associated with Lennox-Gastaut Syndrome.
EMERYVILLE, California, December 20, 2021 — Zogenix (Nasdaq: ZGNX), a global biopharmaceutical company developing and commercializing rare disease therapies, today announced that it has submitted its Type II Variation Market Authorization Application to the European Medicines Agency (EMA) for FINTEPLA® for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), a highly treatment-resistant form of childhood-onset epilepsy. If approved, the application would expand the use of FINTEPLA in Europe beyond Dravet syndrome to include LGS.
“The submission of this Type II Variation is a significant milestone for our FINTEPLA program and furthers our goal of bringing FINTEPLA to LGS patients and families in need,” said Gail Farfel, Ph.D., Executive Vice President and Chief Development Officer of Zogenix. “We remain focused on working with the EMA to bring FINTEPLA for the treatment of seizures associated with LGS to market as quickly as possible in Europe.”
The EMA submission for FINTEPLA is supported by data from a global randomized, placebo-controlled Phase 3 clinical trial Study 1601 in 263 patients (age 2-35 years) that demonstrated FINTEPLA at a dose of 0.7/mg/kg/day was superior to placebo in reducing the frequency of drop seizures (p=0.0012). The same dose of FINTEPLA (0.7 mg/kg/day) also demonstrated statistically significant improvement versus placebo in the key secondary efficacy measure, the proportion of patients with a clinically meaningful reduction (≥50%) in drop seizure frequency. The submission also includes long-term safety and effectiveness data from Zogenix’s on-going open-label extension trials. FINTEPLA has been generally well-tolerated, with the adverse events observed to date consistent with those observed in the Company’s prior Phase 3 studies in Dravet syndrome.
In December 2021, the U.S. Food and Drug Administration (FDA) accepted Zogenix’s supplemental New Drug Application (sNDA) and granted Priority Review for FINTEPLA for the treatment of seizures associated with LGS. FINTEPLA is approved by the FDA and European Commission for the treatment of seizures associated with Dravet syndrome, a rare infant- and childhood-onset epilepsy marked by frequent and severe treatment-resistant seizures, in patients 2 years of age and older. The Japanese Ministry of Health, Labour & Welfare (MHLW) has also granted Orphan Drug Designation to FINTEPLA for the treatment of seizures associated with Dravet syndrome, which Zogenix is developing in Japan.
About Lennox-Gastaut Syndrome
Lennox-Gastaut Syndrome (LGS) is a rare, highly-refractory and devastating lifelong childhood-onset epilepsy that can arise from multiple different causes. There are an estimated 20,000-30,000 LGS patients in the EU.¹ The vast majority of patients do not have well-controlled seizures, despite a regimen of two to five antiepileptic drugs.¹ LGS is characterized by many different seizure types, including many that result in frequent falls and injuries and that often don’t respond to currently available seizure medications. The intellectual and behavioral problems associated with LGS, as well as around-the-clock care requirements, add to the complexity of life with this disease.
Zogenix is a global biopharmaceutical company committed to developing and commercializing therapies with the potential to transform the lives of patients and their families living with rare diseases. The company’s first rare disease therapy, FINTEPLA® (fenfluramine) oral solution, has been approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency and is in development in Japan for the treatment of seizures associated with Dravet syndrome, a rare, severe lifelong epilepsy. The U.S. FDA recently accepted for filing Zogenix’s supplemental New Drug Application (sNDA) and granted Priority Review for the use of FINTEPLA for the treatment of seizures associated with an additional rare epilepsy, Lennox-Gastaut syndrome (LGS). Zogenix also plans to initiate a study of FINTEPLA in a genetic epilepsy called CDKL5 Deficiency Disorder (CDD) and is collaborating with Tevard Biosciences to identify and develop potential next-generation gene therapies for Dravet syndrome and other genetic epilepsies. The company has an additional late-stage development programs, MT-1621, in a mitochondrial disease called TK2 deficiency.
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 potential for Zogenix’s product candidates to provide new treatment options. 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: FINTEPLA may not achieve broad market acceptance as a treatment option of Dravet syndrome which would limit Zogenix’s ability to general revenues; Zogenix may not be successful in executing its sales and marketing strategy for the commercialization of FINTEPLA in the U.S. and Europe, including due to the costs and procedures related to the REMS certification process or controlled access program; the COVID-19 pandemic may continue to disrupt Zogenix’s business operations, impairing the ability to commercialize FINTEPLA in Europe and Zogenix’s ability to generate product revenue in Europe and conduct its development programs; unexpected adverse side effects or inadequate therapeutic efficacy of fenfluramine that could limit regulatory approval or commercialization, or that could result in recalls or product liability claims; later developments with FDA that may be inconsistent with the already completed meetings; additional data from Zogenix’s ongoing studies may contradict or undermine the data previously reported; the potential for the FDA to delay timing of review of the sNDA due to the FDA’s internal resource constraints or other reasons; 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.
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1 Zogenix estimates.