According to a story from pm360online.com, the drug developer Zynerba Pharmaceuticals, Inc., recently announced the release of data from an open label, 12 month study of the company’s experimental product ZYN002. In this long term trial, the drug was used as a treatment for behavioral and emotional symptoms associated with the rare disorder fragile X syndrome. Zynerba is dedicated to the development of transdermal cannabinoid based therapy for rare neuropsychiatric disorders.
About Fragile X Syndrome
Fragile X syndrome is a genetic disorder which affects men more often than women. It is characterized by intellectual disability and distinctive physical features. The disease is caused by a mutation affecting the FMR1 gene. Symptoms of fragile x syndrome include intellectual disability which can vary widely in severity, autism, social anxiety, ADHD, deficits of working memory, seizures, vision problems, and hyperactive behavior. Distinct physical characteristics become noticeable once puberty begins and may include prominent ears, poor muscle tone, a long face, enlarged testicles, soft skin, flat feet, and hyperflexible finger joints. Treatment is mostly symptomatic and may include antidepressants, antipsychotics, and stimulants. Interventions such as special education, speech therapy, and behavioral therapy can be beneficial. There is no cure for fragile X syndrome. Life expectancy is about 12 years lower than the general population. To learn more about fragile X syndrome, click here.
The study found that ZYN002, which is a CBD gel designed for absorption through the skin, was able to have a positive impact on emotional and behavioral symptoms of fragile X syndrome that was sustained throughout the 12 month trial period. Behaviors and emotional problems such as anxiety, compulsive behavior, hyperactivity, social avoidance, and irritability all saw sustained improvement through the course of the trial.
The safety profile of ZYN002 was also favorable; the most common adverse events were upper respiratory tract infection and gastroenteritis, but only 12 percent and 14 percent of participants experienced these events respectively.
Social problems, particularly anxiety and panic attacks, can be a major challenge for boys with fragile X syndrome. These findings suggest that further study of ZYN002 as a treatment for these symptoms is warranted.