Zynerba Pharmaceuticals Reports ZYN002 Granted Orphan Drug Designation for Fragile X Syndrome

Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE) announced that the US Food and Drug Association has granted orphan-drug designation to ZYN002 cannabidiol (CBD) gel, for the treatment of Fragile X syndrome (FXS). Fragile X syndrome is a genetic condition that causes intellectual disability, anxiety disorders, behavioral and learning challenges and various physical characteristics.
According to the company news:

CBD has been shown to reduce the metabolism of two endocannabinoids (2-AG and  anandamide) in laboratory studies. The reduction in metabolism increases 2-AG and anandamide concentrations, which modulates neurotransmitter release and restores endogenous stimulation of endocannabinoid receptors. In mouse knockout models for FXS, symptoms are improved when endocannabinoid levels are increased. There is also anecdotal evidence that high levels of CBD oil from plants have proven effective at improving socialization and language skills in children.

Armando Anido, Chairman and CEO of Zynerba Pharmaceuticals commented:

Fragile X syndrome is a rare but debilitating genetic condition for which there are no effective treatment options. By granting orphan drug designation to this promising synthetic CBD compound, the FDA recognizes the significant need for new therapies, and we are working with key opinion leaders to advance ZYN002 CBD Gel into a Phase 2 clinical study in the second half of this year.

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