Zygel (ZYN002 CBD gel) has been granted fast track designation by the United States’ Food and Drug Administration (FDA) for the treatment of behavioral symptoms in patients with fragile X syndrome, its developer Zynerba Pharmaceuticals announced.
A fast track designation is granted to a newly developed medication that has the potential to address an unmet medical need for patients with a serious or fatal disease. The designation allows the FDA to conduct an expedited review of the therapy so that it can reach the market quickly and the patient earlier.
“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,” Armando Anido, chairman and CEO of Zynerba, said in a press release.
Individuals with fragile X can experience significant social and behavioral issues including, but not limited to, social isolation, avoidance of new social activities, irritability, aggressiveness towards others, lack of attention and movement, and resistance towards any physical contact.
Recent studies have suggested that behavioral abnormalities in patients with fragile X and other neurological conditions are associated with a disruption in the endocannabinoid system which is thought to be modulated by cannabidiol (CBD) — a naturally occurring chemical found in cannabis that does not get users “high.”
Researchers believe that CBD has the potential to improve behavioral and social symptoms in patients with fragile X.
Zygel is the first CBD formulated clear gel that was manufactured pharmaceutically to deliver controlled amounts of CBD into the bloodstream through the skin.
Currently, Zynerba is exploring Zygel’s potential, against a placebo, in the ongoing Phase 2/3 CONNECT-FX trial (NCT03614663). The study is currently recruiting fragile X syndrome patients, ages 3–17, at clinical sites in Australia, New Zealand, and the United States.
The company expects to announce top line results from CONNECT-FX during the second half of 2019.
“We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome, and we look forward to working closely with the FDA to obtain approval to market Zygel as soon as possible,” Anido added.
Results from a prior Phase 2 trial (FAB-C) showed that one year of treatment with Zygel can help provide sustained benefits in social and behavioral symptoms in young patients with fragile X syndrome.