-=Zogenix (ZGNX) receives refusal to file letter for its seizure drug

Zogenix receives Refusal to File Letter from FDA for FINTEPLA New Drug Application treating seizures associated with Dravet syndrome

Co received a Refusal to File (RTF) letter from the FDA regarding its New Drug Application (NDA) for FINTEPLA (ZX008, fenfluramine hydrochloride) for the treatment of seizures associated with Dravet syndrome.

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.

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.

Zogenix (ZGNX) : positive results for the treatment of Dravet syndrome

• 9/27:  #15;  vol.  900K

Zogenix reports positive top-line results from its first Phase 3 trial for its investigational drug, ZX008 for the treatment of Dravet syndrome; trial met its primary endpoint

"Dravet syndrome is a rare, but catastrophic form of epilepsy that can be devastating for patients and their families," said Joseph Sullivan M.D., director of the Pediatric Epilepsy Center in UCSF Benioff Children's Hospital San Francisco, and Principal Investigator of Study 1 in the U.S. "These results are truly exciting and demonstrate, in a large multicenter controlled trial, the impressive efficacy of low-dose fenfluramine for patients with Dravet syndrome. If approved, ZX008 could play an important role in treating this devastating condition."

• The trial met its primary objective of demonstrating that ZX008, at a dose of 0.8 mg/kg/day, is superior to placebo as adjunctive therapy in the treatment of Dravet syndrome in children and young adults based on change in the frequency of convulsive seizures between the 6-week baseline observation period and the 14-week treatment period. ZX008 0.8 mg/kg/day also demonstrated statistically significant improvements versus placebo in all key secondary measures, including the proportion of patients with clinically meaningful reductions in seizure frequency and longest seizure-free interval. The same analyses comparing a 0.2 mg/kg/day ZX008 dose versus placebo also demonstrated statistically significant improvement compared with placebo.
  
• A key secondary endpoint was the same analysis for a comparison of ZX008 0.2 mg/kg/day and placebo. Patients taking ZX008 0.2 mg/kg/day achieved a reduction in mean monthly convulsive seizures of 33.7% compared to placebo (p=0.019). Collectively, these top-line data suggest a dose-response relationship for ZX008 in the adjunctive treatment of convulsive seizures in Dravet syndrome.

** charts before  **

 

** daily **

** monthly **

*****

GW Pharma (GWPH) down 10% after competitor Zogenix (ZGNX) reported positive Phase 3 data for Dravet syndrome (severe myoclonic epilepsy of infancy)

• In August, GWPH said its NDA rolling submission for Dravet and LGS epilepsy indications was under way.

 

Zogenix (ZGNX)

• Stock: ZGNX (NASDAQ)
• Headquarters: Emeryville, CA
• CEO: Stephen J. Farr
• Founded: 2006
• zogenix.com

  

Zogenix, Inc. is a pharmaceutical company engaged in developing and commercializing central nervous system (CNS) therapies that address specific clinical needs for people living with orphan and other CNS disorders. Its primary area of therapeutic focus is epilepsy and related seizure disorders. Its lead product candidate, ZX008, is a low-dose fenfluramine for the treatment of seizures associated with Dravet syndrome. ZX008 has received orphan drug designation in the United States and European Union (EU), for the treatment of Dravet syndrome. The Company has an additional product candidate, Relday (risperidone once-monthly long-acting injectable) for the treatment of schizophrenia. Relday is a long-acting injectable formulation of risperidone. Risperidone is used to treat the symptoms of schizophrenia and bipolar disorder in adults and teenagers 13 years of age and older. It has completed the Phase I program for Relday.

Key stats and ratios

	Q2 (Jun '17)	2016
Net profit margin	-315.13%	-238.08%
Operating margin	-309.84%	-251.94%
EBITD margin	-	-217.86%
Return on average assets	-45.10%	-25.57%
Return on average equity	-99.73%	-45.26%
Employees	67