Zogenix Announces U.S. FDA Acceptance for Priority Review of Supplemental New Drug Application for FINTEPLA® (Fenfluramine) for the Treatment of Seizures Associated with Lennox-Gastaut Syndrome (LGS)
Zogenix (NASDAQ: ZGNX) announced that the FDA accepted for filing its supplemental New Drug Application (sNDA) for FINTEPLA for treating seizures associated with Lennox-Gastaut syndrome (LGS), granting Priority Review. The PDUFA target action date is set for March 25, 2022. The sNDA is based on a positive Phase 3 trial showing FINTEPLA significantly reduced drop seizures in patients aged 2-35. Zogenix previously received FDA approval for FINTEPLA for Dravet syndrome. LGS affects 30,000 to 50,000 people in the U.S.
- FDA granted Priority Review for FINTEPLA's sNDA for LGS, indicating potential market advantage.
- Successful Phase 3 trial demonstrated FINTEPLA's efficacy in reducing drop seizures, showing a p-value of 0.0012.
- FDA may delay the PDUFA action date or require more data, impacting approval timelines.
- Potential for insufficient market acceptance could limit revenue generation.
Prescription Drug User Fee Act (PDUFA) Target Action Date of March 25, 2022
EMERYVILLE, Calif., Dec. 01, 2021 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies today announced the U.S. Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to the company’s supplemental New Drug Application (sNDA) for the use of FINTEPLA® for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS). The FDA granted Priority Review, which means that the FDA has a goal to complete its review within six months from the date of receipt, with a Prescription Drug User Fee Act (PDUFA) target action date of March 25, 2022. FDA Priority Review is granted for investigational therapies that, if approved, may offer significant improvements in the treatment, prevention or diagnosis of a serious condition.
“This is a critically important milestone for our FINTEPLA development program in LGS and brings us one step closer to a potential new treatment option for this rare and difficult to treat childhood developmental and epileptic encephalopathy,” said Gail Farfel, Ph.D., Executive Vice President and Chief Development Officer of Zogenix. “We look forward to working closely with the FDA to potentially bring FINTEPLA for the treatment of seizures associated with LGS to market as quickly as possible.”
The sNDA submission is based on a positive, 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), as well as long-term safety and effectiveness data from Zogenix’s ongoing open-label extension trials.
In June 2020, FINTEPLA was approved by the FDA for the treatment of seizures associated with Dravet syndrome in patients two years of age and older.
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. There are an estimated 30,000 to 50,000 people who have LGS in the United States.1
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 for the treatment of seizures associated with Dravet syndrome in patients two years of age and older and the European Medicines Agency 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. In the U.S. and Europe, treatment with FINTEPLA is initiated and supervised by physicians with experience in the treatment of Dravet syndrome under a Risk Evaluation Mitigation Strategy (REMS) program (U.S.) or Controlled Access Program (EU). In the United States, please see important prescribing and safety information at www.Fintepla.com. In Europe, please see important prescribing and safety information at www.Fintepla.eu.
In Japan, Zogenix is on track to submit a new drug application (J-NDA) for FINTEPLA for the treatment of seizures associated with Dravet syndrome in late 2021. The company has two additional late-stage development programs: one in a rare epilepsy called Lennox-Gastaut syndrome and one 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.
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 timing of review by the FDA of the sNDA for the use of FINTEPLA for the treatment of seizures associated with LGS; plans to submit a J-NDA for FINTEPLA in Japan and the estimated timing for that submission; the potential for Zogenix’s product candidates to provide new treatment options; and Zogenix’s plans with respect to its development programs. 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 delay the PDUFA target action date related to the LGS sNDA due to the FDA’s internal resource constraints or other reasons; the FDA may disagree that the existing safety and efficacy data is sufficient to approve the sNDA; FINTEPLA may not achieve broad market acceptance as a treatment option of seizures associated with LGS or Dravet syndrome, which would limit Zogenix’s ability to generate revenues; 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; and other risks described in Zogenix’s prior press releases as well as in public periodic filings with the U.S. Securities and 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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Investors
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Media
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1 Zogenix estimates
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