Zynerba Pharma Zygel gets FDA fast track designation for Fragile X
The FDA granted Zynerba Pharma’s (NASDAQ:ZYNE) Zygel fast track designation for behavioral symptoms associated with Fragile X syndrome.
Fragile X syndrome, a rare genetic condition caused by a mutation in the X chromosome’s FMR1 gene, is the leading known cause of inherited intellectual disability and autism spectrum disorder. The mutation causes dysregulation of the endocannabinoid pathway, resulting in mental disabilities and behavioral symptoms.
Zygel in a cannabidiol-based gel that provides controlled drug delivery into the bloodstream through the skin. The CONNECT-FX pivotal trial is testing Zygel in children and adolescents with Fragile X.
“We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome,” Armando Anido, Zynerba’s chairman and CEO, said in a statement.
Zynerba expects to present top-line data from the CONNECT-FX study in the second half of 2019.