Zogenix Receives Positive CHMP Opinion for FINTEPLA® (Fenfluramine) Oral Solution for the Treatment of Seizures in Patients with Dravet Syndrome
- Dravet syndrome is a rare, life-long, infant- and childhood-onset epilepsy associated with severe, treatment-resistant seizures
- CHMP positive opinion based on Phase 3 study data that demonstrated FINTEPLA® significantly reduced convulsive seizure frequency in patients whose seizures were not adequately controlled on other medications, including stiripentol
- Final decision on Marketing Authorization Application expected by year-end 2020
“We are pleased that the CHMP’s regulatory review of FINTEPLA for quality, safety, and efficacy has resulted in their positive opinion,” said
“Reducing seizure frequency is the first and most important step in treating all Dravet syndrome children, regardless of age,” said
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
Earlier this year, FINTEPLA was approved by the
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
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.
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
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.
7 Villas N, Meskis MA, Goodliffe S. Dravet syndrome: characteristics, comorbidities, and caregiver concerns. Epilepsy Behav. 2017;74:81-86.
Source: Zogenix, Inc