Saturday, October 17, 2020
Zogenix, a global biopharmaceutical company developing rare disease therapies, announced that the Committee for Medicinal Products for Human Use (CHMP), a part of the European Medicines Agency (EMA), has adopted a positive opinion recommending the marketing authorization of FINTEPLA® (fenfluramine) oral solution for the treatment of seizures associated with Dravet syndrome, a rare and devastating infant- and childhood onset epilepsy, as an add-on therapy to other antiepileptic medicines for patients two years of age and older. The European Commission (EC) is expected to make a final decision on the company’s Marketing Authorization Application (MAA) by the end of the year.
“We are pleased that the CHMP’s regulatory review of FINTEPLA for quality, safety, and efficacy has resulted in their positive opinion,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix. “We began our rigorous global development program for FINTEPLA nearly six years ago after researchers in Belgium recognized the potential of fenfluramine, a drug with distinct pharmacology from all other anticonvulsant agents, to treat intractable seizures in Dravet syndrome. Many Dravet syndrome patients continue to experience frequent severe seizures even while taking one or more currently available anti-seizure medications. For this reason, we are excited to be another step closer to potentially introducing FINTEPLA as an important new treatment option for these patients and their families in Europe.”
“Reducing seizure frequency is the first and most important step in treating all Dravet syndrome children, regardless of age,” said Lieven Lagae, M.D, Ph.D., Full Professor and Head of Pediatric Neurology Department at the University Hospitals of Leuven in Belgium. “I am thrilled that all Phase 3 studies with fenfluramine demonstrated a clinically meaningful, highly statistically significant decrease of seizure frequency in Dravet syndrome patients.”
The MAA for FINTEPLA included positive results from two randomized, controlled Phase 3 trials (Study 1 and Study 2), together with an interim analysis of an ongoing long-term, open-label extension study involving a total of 330 Dravet syndrome patients. These studies demonstrated that adjunctive fenfluramine treatment provided a highly statistically significant and clinically meaningful reduction in convulsive seizure frequency compared to placebo and was generally well-tolerated. In one of the trials, Study 2, all subjects were treated with a background regimen that included stiripentol, with significant improvement observed for FINTEPLA over placebo. The long-term, open-label extension study demonstrated durable efficacy, with patients in that study treated for up to three years with FINTEPLA. The most commonly reported adverse events experienced during these studies were decreased appetite, diarrhea, pyrexia, fatigue, upper respiratory tract infection, lethargy, somnolence and bronchitis.1,2,3 No patient developed any cardiovascular adverse events, including valvular heart disease or pulmonary arterial hypertension.
If authorized by the EC, FINTEPLA will be approved for use by patients with Dravet syndrome aged two years and older in all European Union member states, as well as the United Kingdom, Iceland, Liechtenstein and Norway. The product is expected to be made available under a controlled access program to ensure regular cardiac monitoring and to mitigate potential off-label use for weight management.
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. A third positive Phase 3 clinical trial (Study 3) was recently reported to support registration of FINTEPLA in Japan.
About Dravet Syndrome
Dravet syndrome is a rare, devastating and life-long form of epilepsy that 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. Affecting one in 15,700 individuals in the U.S. and approximately one in 20,000 to 40,000 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, and financial burden associated with the disease.4,5,6,7
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 has received a positive CHMP opinion in Europe for the treatment of seizures associated with Dravet syndrome, a rare, severe childhood onset epilepsy in patients aged two and older. The company has two additional late-stage development programs underway: one for FINTEPLA for the treatment of seizures associated with Lennox-Gastaut syndrome, a different rare childhood-onset epilepsy and another for MT1621, an investigational novel substrate enhancement therapy for the treatment of TK2 deficiency, a rare genetic disorder. MT1621 is being developed through Modis Therapeutics, a Zogenix company.
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 FINTEPLA, if approved by the EC, will be an important new treatment option for Dravet syndrome patients; and the timing and results of any decision regarding the MAA for FINTEPLA for the treatment of seizures associated with Dravet syndrome. 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 EC may not agree with the Company’s interpretation of the clinical data submitted in the MAA; the EC may not affirm the CHMP opinion and grant a centralized marketing authorization; additional data from Zogenix’s ongoing studies may contradict or undermine the data submitted in the Dravet syndrome MAA for FINTEPLA or reported for LGS; 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.
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1 Lagae L, Sullivan J, Knupp K, et al. Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial. Lancet. 2020;394:2243-2254.
2 Nabbout R, Mistry A, Zuberi S, et al. Fenfluramine for treatment-resistant seizures in patients with Dravet syndrome receiving stiripentol-inclusive regimens. JAMA Neurol. 2020;77:300-308.
3 Sullivan J, Auvin S, Pringsheim M, et al. Long-term (2-year) safety and efficacy of adjunctive ZX008 (fenfluramine hydrochloride oral solution) for Dravet syndrome: Interim results of an ongoing open-label extension study. Originally scheduled for presentation at the cancelled American Academy of Neurology 2020 Annual Meeting. Program number S31.007.
4 Aras LM, Isla J, Mingorance-Le Meur A. The European patient with Dravet syndrome: results from a parent-reported survey on antiepileptic drug use in the European population with Dravet syndrome. Epilepsy Behav. 2015;44:104-9.
5 Lagae L, Brambilla I, Mingorance A, et al. Quality of life and comorbidities associated with Dravet syndrome severity: a multinational cohort survey. Dev Med Child Neurol. 2018;60:63-72.
6 Sullivan J, Knupp K, Wirrel E. Rare disease database: Dravet syndrome. National Organization of Rare Diseases. Available at: https://rarediseases.org/rare-diseases/dravet-syndrome-spectrum/. Last updated: 2018. Last accessed: September 2020.
7 Villas N, Meskis MA, Goodliffe S. Dravet syndrome: characteristics, comorbidities, and caregiver concerns. Epilepsy Behav. 2017;74:81-86.