The Food and Drug Administration (FDA) has granted Fast Track status to OV101 (gaboxadol; Ovid Therapeutics), a delta (δ)-selective GABAA 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.
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.
For more information visit OvidRx.com.
This article originally appeared on MPR