DEVON, PA — Zynerba Pharmaceuticals, Inc. (NASDAQ: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, provided an update on its Fragile X syndrome (FXS) and developmental and epileptic encephalopathies (DEE) programs.
Fragile X syndrome (FXS)
Zynerba has been notified that the U.S. Food and Drug Administration (FDA) will meet with the Company via teleconference in the fourth quarter of 2020 to discuss the pivotal CONNECT-FX data and the regulatory path forward in patients with FXS and a fully methylated FMR1 gene (FMet). The Company also expects to disclose the outcome of the meeting in the fourth quarter of this year.
“The meeting with the FDA will be an important milestone for patients and their families who live with the debilitating behavioral impact of Fragile X,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba.
“Our ongoing evaluation of the pivotal CONNECT-FX data continues to clarify the impact that Zygel achieved in the most severely impacted children and adolescents with FXS, as well as the excellent tolerability profile. We look forward to discussing the pivotal data and the regulatory path for potential approval in FMet patients with the FDA in the fourth quarter of this year.”
Zynerba also announced that the U.S. Patent and Trademark Office has issued US Patent No. 10,758,497, titled “Treatment of Fragile X Syndrome with Cannabidiol” which includes claims directed to a method of treating FXS comprising administering 250mg or 500mg of synthetic or purified cannabidiol in a pharmaceutically acceptable carrier to a person in need thereof. This new patent, which expires in 2038, is part of an expanding intellectual property portfolio covering Zygel.
Developmental and epileptic encephalopathies (DEE)
Zynerba has concluded its iterative meetings with the FDA utilizing their ‘Written Response Only’ (WRO) format to discuss the clinical pathway for Zygel in DEE. The FDA supports a development program that would treat focal-impaired awareness and convulsive seizures.
However, due to the heterogeneity of patients who fall under the DEE umbrella, the FDA suggests that Zynerba pursue individual syndromes rather than considering DEE patients as a single disorder or condition. The Company is in the process of finalizing its evaluation of which epileptic syndromes it may pursue with Zygel.
“We appreciate our partnership with the FDA, and thank them for their input and support as we seek to advance the development of Zygel in certain rare epilepsy syndromes,” continued Anido. “We look forward to completing our target assessments and communicating our path forward around the end of this year.”
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