Zogenix Submits Supplemental New Drug Application for FINTEPLA® (Fenfluramine) for the ...
- Supplemental New Drug Application (sNDA) submission is supported by existing clinical data, including positive data from Company’s Phase 3 trial, Study 1601, in which the primary endpoint was met with high statistical significance.
- Lennox-Gastaut Syndrome (LGS) is estimated to affect approximately 30,000-50,000 patients in the U.S.¹
EMERYVILLE, Calif., Sept. 28, 2021 (GLOBE NEWSWIRE) -- Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that it has submitted a supplemental New Drug Application (sNDA) for FINTEPLA® (fenfluramine) for the treatment of seizures associated with Lennox-Gastaut Syndrome (LGS) to the U.S. Food and Drug Administration (FDA).
“As LGS is a severe, rare form of epilepsy that is not well-controlled by currently available anti-seizure medications, we believe that FINTEPLA, if approved, would become an important new treatment option that addresses a significant unmet need for this patient population,” said Gail Farfel, Ph.D., Executive Vice President and Chief Development Officer of Zogenix. “We would like to thank the LGS patients, families, and healthcare providers who worked tirelessly alongside us to achieve this milestone in FINTEPLA’s development. We appreciate the FDA’s guidance through the submission process and look forward to continuing to work closely with the Agency during their review of our application.”
The sNDA 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.
LGS is a rare and highly refractory form of childhood-onset epilepsy that is difficult to treat. Patients suffer from significant intellectual, behavioral, and motor disabilities, and have a high risk of status epilepticus or sudden unexpected death in epilepsy. There are an estimated 30,000-50,000 LGS patients in the U.S.¹ The vast majority of patients do not have well-controlled seizures, despite a regimen of two to five antiepileptic drugs¹.
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 and devastating lifelong childhood-onset epilepsy that can arise from multiple different causes. 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. FDA and the European Medicines Agency and is in development in Japan for the treatment of seizures associated with Dravet syndrome and LGS, both rare, severe lifelong epilepsies. The company has one additional late-stage development program: in a mitochondrial disease called TK2 deficiency. 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.
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 FINTEPLA to reduce the frequency of seizures and provide clinical benefit to LGS patients, if approved, and statements regarding Zogenix’s clinical development plans. 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 issue a “refuse to file” letter with respect to our sNDA submission if the FDA does not believe the sNDA is ready for review; the potential for the FDA to delay timing of review of the sNDA due to the FDA’s internal resource constraints or other reasons; interim data from Part 2 of Study 1601 and other clinical trials may change as more patient data become available and could result in material changes in the final data, impairing regulatory submissions and approvals; unexpected adverse side effects or inadequate therapeutic efficacy of fenfluramine that could limit approval in Japan or commercialization in the United states or Europe, 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 Zogenix estimates.