Zynerba Pharmaceuticals Provides Regulatory Update on Zygel in Fragile X Syndrome
- Single Trial to be Conducted in Patients with Fragile X Syndrome to Confirm Positive Results Seen in the Population of Responders in the CONNECT-FX Trial -
- Conference Call and Webcast Today, December 17, 2020 at 8:30 am ET -
DEVON, Pa., Dec. 17, 2020 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided an update on its meeting with the U.S. Food and Drug Administration (FDA) regarding its Fragile X syndrome (FXS) program. The Company plans to conduct a double-blind, placebo-controlled pivotal trial in patients with FXS who have a highly methylated FMR1 gene to confirm the positive results observed in this population of responders in the CONNECT-FX trial. In the first half of 2021, Zynerba will review the trial design and protocol for the new trial through a Type C meeting with the FDA and expects to initiate the pivotal trial before the end of 2021. Zynerba believes that positive results from this confirmatory trial would be sufficient to support the submission of a New Drug Application (NDA) for Zygel in FXS.
“We believe that Zygel has the potential to meaningfully relieve the behavioral symptoms of the most impacted individuals with Fragile X syndrome. We are committed to bringing this important therapy to patients and their families within the Fragile X community,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We are thankful for our ongoing constructive dialogue with the FDA on our path forward to NDA submission. Completing the development of Zygel in FXS and preparing for a successful launch will be the primary focus of the Company.”
“The results reported in the analysis of children with a highly methylated FMR1 gene are a source of considerable hope for the patients and their families who are impacted by Fragile X syndrome,” said Linda Sorensen, Executive Director of the National Fragile X Foundation (NFXF). “Zynerba has been an important partner to the families of children with Fragile X. We thank them for their continued dedication and commitment to bringing an FDA-approved treatment to our families and look forward to helping with the recruitment of patients for this study.”