Zogenix Receives Refusal to File Letter from U.S. Food and Drug Administration for Fintepla New
Drug Application
EMERYVILLE, Calif., April 08, 2019 (GLOBE NEWSWIRE) -- Zogenix, Inc. (NASDAQ: ZGNX), a global pharmaceutical company developing rare disease therapies, today announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (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.
“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.
About Zogenix
Zogenix is a global pharmaceutical company committed to developing and commercializing transformative therapies to improve the lives of patients and their families living with rare diseases. The company’s lead candidate, Fintepla® (ZX008, fenfluramine) has been accepted for review by the European Medicines Agency and is in development in Japan. For
Many parents of lesbian, gay or bisexual (LGB) children take years to adjust after learning about their sexual orientation, a new study finds.
ReplyDeleteThe study included more than 1,200 parents of LGB youth aged 10 to 25. The parents visited a website with LGB resources and were asked to complete a questionnaire.
Of those parents, 26% had learned that their child identified as LGB in the past month.
The parents were asked: "How hard is it for you, knowing that your son or daughter is gay, lesbian or bisexual?" They responded on a five-point scale, ranging from not at all hard to extremely hard.
The survey revealed that adjusting takes time. Parents who had learned about their child's sexual orientation two years ago struggled just as much as those who had recently been told.
Black and Hispanic parents reported a harder adjustment than white parents, and parents of older youth had greater difficulty than parents of younger children, the findings showed.
DeleteFathers and mothers had similar levels of difficulty, as did parents of boys and girls, according to the study published June 18 in the journal Archives of Sexual Behavior.
Parents who'd known for five years or more that their child is LGB reported the least amount of difficulty.
"Surprisingly, we found that parents who knew about a child's sexual orientation for two years struggled as much as parents who had recently learned the news," said David Huebner, associate professor of prevention and community health at George Washington University, in Washington, D.C. "Two years is a very long time in the life of a child who is faced with the stress of a disapproving or rejecting parent."
Previous research suggests that parents who have trouble adjusting are more likely to disapprove or behave negatively, which can put LGB youth at risk of serious health problems.
One reason parents may have trouble accepting that their child is LGB is concern that he or she may face bullying or harassment, and a harder life overall, Huebner suggested.