OV101 Wins Orphan Drug Status for Rare Neurological Disorder

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The treatment is currently being investigated in a Phase 2 trial in adults with Angelman syndrome

The Food and Drug Administration (FDA) has granted Orphan Drug designation to Ovid Therapeutics’ OV101 for the treatment of Angelman syndrome, a rare, genetic disorder that causes developmental and neurologic disabilities.

The pharmaceutical company is currently investigating OV101 in the STARS study, a prospective, randomized, double-blind, placebo-controlled Phase 2 trial designed to explore the efficacy endpoints and evaluate the safety parameters of OV101 in adult patients with Angelman syndrome.

OV101 is believed to be the only clinically tested delta (δ)-selective GABA-A receptor agonist under development to normalize tonic inhibition in patients with Angelman syndrome. It also has the potential to enhance neuronal function, influence sleep, motor control,cognition, and improve outcomes in patients living with the disease.


Ovid Therapeutics Receives Orphan Drug Designation from the US FDA for OV101 for the Treatment of Patients with Angelman Syndrome [press release]. New York, NY: Ovid Therapeutics newsroom; September 8, 2016. 

This article originally appeared on MPR