FDA Grants Orphan Drug Status to OV101 for Rare, Genetic Neurologic Disorder

The treatment is currently being investigated in a Phase 2 trial in adults with Angelman syndrome
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 OV101 (Ovid Therapeutics) for the treatment of Angelman syndrome, a rare, genetic disorder that causes developmental and neurologic disabilities.

The 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. 

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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 and cognition, and improve outcomes in patients living with the disease.

For more information visit Ovidrx.com.

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