Fast Track designation has been granted to OV101 (gaboxadol, Ovid Therapeutics) for the potential treatment of Angelman syndrome.

Gaboxadol is a delta (δ)-selective GABAA receptor agonist that targets the disruption of tonic inhibition, a central physiological process of the brain thought to be the underlying cause of Angelman syndrome. It is the first investigational treatment to target tonic inhibition, a process thought to be the underlying cause of certain neurodevelopmental disorders. 

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A Phase 2 trial, ‘STARS’, is currently investigating the safety and efficacy of OV101. A Phase 1 trial established that gaboxadol has a similar pharmacokinetic profile in adolescents as in adults. In total, 4,000 patients have been administered gaboxadol in testing with favorable safety profiles observed.

Angelman syndrome affects approximately 1 in 12,000 to 20,000 in the U.S. and symptoms include delayed development, intellectual disability, and seizures. There is currently no FDA-approved treatment for this disorder.

“We believe that OV101, with its novel mechanism of action, has the potential to be an innovative and impactful therapy,” said Amit Rakhit, MD, MBA, CMO of Ovid Therapeutics. “We look forward to data from the STARS trial in the second half of 2018.”

The Company has also received Orphan Drug designation for gaboxadol in the treatment of Fragile X syndrome.

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