An Experimental Treatment for Fragile X and Angelman Syndromes Gets Fast Track Designation

According to a story from, the pharmaceutical company Ovid Therapeutics announced recently that the U.S. Food and Drug Administration (FDA) has given its drug candidate OV101 Fast Track designation for the treatment of fragile X syndrome. Ovid Therapeutics specializes in the development of therapies that will offer transformative improvements for the lives of people with rare neurological conditions.
Fragile X syndrome (FXS) is a genetic disorder that causes intellectual disability. Generally, boys are more affected by the syndrome that girls. Along with intellectual problems, patients display a specific combination of physical features, such as a long face, prominent ears, flexible fingers and double-jointed thumbs, flat feet, low muscles tone, and large testicles. Fragile X syndrome often occurs alongside autism and is often considered the cause of autism in such cases. ADHD is also common in patients with fragile X, and patients often have much shorter than average attention spans. Fragile X patients often experience strong social anxiety. There are currently no approval treatments for the condition, so most treatment options focus on alleviating symptoms and complications like ADHD. Therapy can also provide significant benefits, and early intervention is strongly recommended. To learn more about fragile X syndrome, click here.

The FDA’s Fast Track designation is reserved for investigational therapies that have demonstrated the potential to meet a medical need for a debilitating, life threatening, or otherwise medically serious condition that is not being fulfilled by currently available medications. The designation will allow Ovid to be able to communicate more frequently with the agency during the clinical trials process. Generally, medications with Fast Track designation will be evaluated within a 6 month time frame instead of the standard 10 month time frame.

Ovid plans to begin Phase II trials of OV101 later this year, and will test the drug for the treatment of 13 to 22 year old males with FXS. The earlier Phase I trials tested the drug as a treatment for both FXS and Angelman syndrome. A Phase II trial for Angelman syndrome is currently scheduled to begin in late 2018. If the trials go as planned, this new treatment has the potential to improve the lives of patients with these rare disorders.

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