Experimental Therapy for Fragile X Syndrome Earns Fast Track Designation

According to a story from globenewswire.com, the drug developer Zynerba Pharmaceuticals recently announced that the company has been awarded Fast Track Designation from the US Food and Drug Administration (FDA). This designation is in regards to the company’s investigational product candidate Zygel, which is being tested as a treatment for behavioral symptoms associated with fragile X syndrome, a rare genetic disorder. Zynerba is focused on the development of cannabinoid-based transdermal therapies 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.

Fast Track Designation

Fast Track Designation is an FDA program that is designed with the intent of accelerating the review process for therapies that have the potential to treat serious, life-threatening diseases. To qualify, a drug must display the potential to fulfill a medical need that is not being met by current therapies and must be designed to treat a life-threatening disease. Benefits of the Fast Track Designation include close collaboration with agency officials and the potential to qualify for other incentive programs like Priority Review if other relevant criteria are met.

Zygel is a CBD formulated gel that is designed to deliver its active ingredient through the skin. The potential utility of this therapy in treating fragile X syndrome and other similar conditions, such as autism, is grounded in research that suggests that these conditions involve abnormal disruption of the endocannabinoid system. A Phase 2 clinical trial of Zygel for behavioral symptoms associated with fragile X syndrome is currently under way.


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