Zogenix gets refusal to file letter from FDA on Fintepla NDA
Zogenix (ZGNX) announced that it received a Refusal to File, RTF, letter from the U.S. Food and Drug Administration regarding its New Drug Application for FINTEPLA, for the treatment of seizures associated with Dravet syndrome.
Dravet syndrome is a rare, catastrophic, lifelong form of epilepsy that begins in the first year of life with frequent and/or prolonged seizures. Previously known as Severe Myoclonic Epilepsy of Infancy (SMEI), it affects 1:15,700 individuals, 80% of whom have a mutation in their SCN1A gene.
Upon its preliminary review, the FDA determined that the NDA, submitted on February 5, 2019, was not sufficiently complete to permit a substantive review.
In the letter, the FDA cited two reasons for the RTF decision: first, certain non-clinical studies were not submitted to allow assessment of the chronic administration of fenfluramine; and, second, the application contained an incorrect version of a clinical dataset, which prevented the completion of the review process that is necessary to support the filing of the NDA.
The FDA has not requested or recommended additional clinical efficacy or safety studies.
The company will seek immediate guidance, including a Type A meeting with the FDA, to clarify and respond to the issues identified in the RTF letter.
“We remain highly confident in FINTEPLA’s clinical profile demonstrated in the Phase 3 program in Dravet syndrome and are committed to advancing the product candidate as a potential new treatment option for this and other rare and often catastrophic epileptic encephalopathies,” said Stephen J. Farr, Ph.D., President and CEO of Zogenix.
“We are fully committed to working with the FDA as quickly as possible to address the open issues and clarify the path to successfully re-filing our application.”
Zogenix’s Marketing Authorization Application, MAA, for FINTEPLA for the treatment of seizures associated with Dravet syndrome was previously accepted for review by the European Medicines Agency, EMA, and the Company anticipates an approvability decision could be reached by the EMA in the first quarter of 2020.
Piper Jaffray analyst Danielle Brill lowered her price target on Zogenix to $68 after it received a refusal to file letter from FDA on its Fintepla new drug application.
The analyst notes that while the issues mentioned by the FDA sound like a simple fix, if the agency requires the company to conduct new toxicity studies, the re-submission could be delayed another 12-15 months.
In spite of the “discouraging news”, longer term, Brill still keeps her Overweight rating on Zogenix and believes that “the company should be able to leverage existing ICH data with fenfluramine” based on their reported prior FDA interactions, modeling a launch in Q2 of 2020.
ZGNX closed at $51.85, it last traded at $35.00.
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