The Food and Drug Administration (FDA) has granted Fast Track designation to Zygel (ZYN002; Zynerba), a cannabidiol gel for the treatment of behavioral symptoms associated with Fragile X syndrome (FXS).
Fragile X is a rare inherited syndrome of intellectual disability and autism. Mutations occur in the FMR1 gene which can block expression of the Fragile X Mental Retardation Protein (FMRP), an important protein in GABA synthesis.
The safety and efficacy of the transdermal gel will be evaluated in a placebo-controlled study involving 204 patients with FXS. The primary outcome measure of the study will be the change from baseline to end of treatment (Week 14) in the Aberrant Behavior Checklist-Community Fragile X Factor Structure (ABC-C FXS) pre-specified subscale; the ABC-C is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.
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“The FDA’s decision to grant Fast Track Designation for Zygel underscores the significance and severity of the unmet medical need that exists for patients living with Fragile X Syndrome and their caregivers,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome.”
Zygel is also being assessed in 2 separate phase 2 studies, one in children with developmental and epileptic encephalopathy and another in patients with autism spectrum disorder.
For more information visit ConnectFXTrial.com.
This article originally appeared on MPR
