- Dravet syndrome is a rare, debilitating and difficult-to-treat lifelong epilepsy that begins in infancy
- EC approval is based on Phase 3 study data demonstrating that FINTEPLA safely and significantly reduced convulsive seizure frequency for Dravet syndrome patients whose seizures were not adequately controlled on existing medications, including stiripentol
- First EU market launch planned for Germany in Q1 2021
EMERYVILLE, Calif., Dec. 21, 2020 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, announced today that the European Commission (EC) has granted marketing authorization for FINTEPLA® (fenfluramine) oral solution for the treatment of seizures associated with Dravet syndrome as an add-on therapy to other anti-epileptic medicines for patients two years of age and older. Dravet syndrome is a rare, lifelong epilepsy that begins in infancy and is marked by severe refractory seizures, frequent medical emergencies, significant cognitive and behavioral impairments, and an increased risk of sudden premature death (SUDEP).
“We deeply appreciate the physicians and Dravet community whose support led to this important milestone,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix. “With the EC approval in place, we can now begin making FINTEPLA more widely available for the treatment of Dravet syndrome patients in Europe who seek new safe and effective treatment options.”
The EC’s approval of FINTEPLA was based on positive safety and efficacy results from two randomized, international, multi-center, placebo-controlled Phase 3 trials (Study 1 and Study 2), as well as data from an interim analysis of a long-term, open-label extension study in 330 Dravet syndrome patients treated up to 3 years.
“After a healthy start in life, children with Dravet syndrome suffer from frequent and prolonged epileptic seizures, which determine the future of the child and mean that parents are on constant alert. Current treatments for Dravet syndrome are unsatisfactory, resulting in the disease affecting motor and mental development,” said Dr. Tilman Polster, a pediatric epilepsy specialist at the Mara Hospital of the Bethel Epilepsy Centre in Bielefeld, Germany, and primary investigator for fenfluramine oral solution in Dravet syndrome in Germany. “Experience from clinical studies has shown that FINTEPLA offers an impressive reduction in seizures, plus an improvement in quality of life. In conjunction with the ongoing data being collected on the safety profile of the therapy, FINTEPLA represents an effective new treatment option and an important hope for the families concerned.”
When added to other antiepileptic therapies, including stiripentol, FINTEPLA provided a highly statistically significant and clinically meaningful reduction in convulsive seizure frequency. The most commonly reported adverse events that occurred in patients treated with this included decreased appetite, diarrhea, pyrexia, fatigue, upper respiratory tract infection, lethargy, somnolence and bronchitis.
With this approval, and subject to price and reimbursement being implemented according to national regulations, Zogenix will be able to market FINTEPLA in all European Union member countries, and in the United Kingdom, Norway, Iceland, and Liechtenstein.
Controlled Access Program
FINTEPLA will be available in Europe under a controlled access program requested by the European Medicines Agency to prevent off-label use for weight management and to confirm that prescribing physicians have been informed of the need for periodic cardiac monitoring in patients taking FINTEPLA.
Zogenix will also conduct the FINTEPLA Registry, an observational registry to provide data on long-term safety of FINTEPLA and frequency of echocardiographic monitoring in routine practice.
Earlier this year, FINTEPLA was approved by the U.S. Food & Drug Administration (FDA) for the treatment of seizures associated with Dravet syndrome in patients aged two years and older. In addition, Zogenix recently reported positive results of a third Phase 3 study of FINTEPLA in Dravet syndrome to support planned registration in Japan, which corroborated the highly statistically significant and clinically meaningful convulsive seizure reductions seen in earlier multinational Phase 3 studies. FINTEPLA is also being studied for the potential treatment of seizures associated with other rare epilepsies.
About Dravet Syndrome
Dravet syndrome is a rare, devastating and life-long form of epilepsy that generally begins in infancy and is marked by frequent, treatment-resistant seizures, significant developmental, motor, and behavioral impairments, and an increased risk of sudden unexpected death in epilepsy (SUDEP). Affecting one in 15,700 live births in the U.S. and approximately one in 20,000 to 40,000 live births in Europe, most patients follow a course of developmental delay with cognitive, motor and behavioral deficits that persist into adulthood. Dravet syndrome severely impacts quality of life for patients, families and caregivers due to the high physical, emotional, caregiving, and financial burden associated with the disease.
About Zogenix
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. 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 company has two additional late-stage development programs underway: one for FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome, another rare epilepsy, and one for MT1621, an investigational therapy for the treatment of a rare genetic disorder called TK2 deficiency (being developed through its subsidiary Modis Therapeutics). Zogenix is also collaborating with Tevard Biosciences to identify and develop potential next-generation gene therapies for genetic rare epilepsies.
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 potential that fenfluramine oral solution will be an important new treatment option for Dravet syndrome patients; Zogenix’s plans to commercialize fenfluramine in Europe, including the timing of the launch. 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: Zogenix’s ability to successfully launch FINTEPLA, including launching a controlled access program implemented due to risks related to valvular heart disease and pulmonary arterial hypertension, and the conduct of the FINTEPLA Registry, and the timing thereof; the COVID-19 pandemic may disrupt Zogenix’s business operations, impairing the ability to commercialize FINTEPLA in Europe and Zogenix’s ability to generate product revenue in Europe; Zogenix may not be successful in executing its sales and marketing strategy for the commercialization of FINTEPLA in Europe; unexpected adverse side effects or inadequate therapeutic efficacy of fenfluramine that could limit 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.
CONTACTS:
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