The Food and Drug Administration (FDA) has granted Fast Track status to OV101 (gaboxadol; Ovid Therapeutics), a delta (δ)-selective GABA receptor agonist, for the treatment of Fragile X syndrome. 

Fragile X is a rare inherited syndrome of intellectual disability and autism. Mutations occur in the FMR1 gene which can block expression of the Fragile X Mental Retardation Protein (FMRP), an important protein in GABA synthesis.

The Company has completed a Phase 1 safety study in adults and children who have Fragile X and Angelman syndrome, which proved successful. A Phase 2 trial of OV101 in males with Fragile X syndrome aged 13 to 22 is planned for later in 2018. Topline data from the company’s Phase 2 STARS clinical trial investigating OV101 for the treatment of adults and adolescents with Angelman syndrome are expected in the second half of 2018.


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There is currently no FDA-approved treatments for Fragile X syndrome. “We are committed to exploring the potential of OV101 to become a transformative medicine and we look forward to announcing further progress this year,” said Amit Rakhit, MD, MBA, chief medical officer of Ovid. 

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For more information visit OvidRx.com.

This article originally appeared on MPR