Ovid’s OV101 Earns Orphan Drug Designation in Europe to Treat Angelman Syndrome

Patricia Inacio, PhD avatar

by Patricia Inacio, PhD |

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The European Commission (EC) has granted orphan drug designation to Ovid Therapeutics’ OV101 for the treatment of Angelman syndrome. The therapy is being tested in the Phase 3 NEPTUNE trial that soon will begin enrolling participants.

Orphan drug status is intended to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from application fees usually imposed by the EC or the U.S. Food and Drug Administration (FDA).

“We are proud to be granted Orphan Drug Designation by the European Commission. This is another important step and further validates the potential of OV101 to be a meaningful treatment option for those living with Angelman syndrome around the world,” Amit Rakhit, MD, MBA, said in a press release. Rakhit is chief medical officer and head of research and  development at Ovid.

OV101 , also called gaboxadol or THIP, is a small molecule derived from a compound called muscimol, which is naturally found in a mushroom (Amanita muscaria). Given orally, it selectively activates GABA receptors located on the surface of nerve cells outside the synapse — the region between two nerve cells that allows them to communicate — and restores tonic inhibition. That allows the brain to balance different types of neurological signals generated by nerve cells.

The EC’s decision was based on promising results from the Phase 2 STARS clinical trial (NCT02996305), in which treatment with OV101 improved sleep, behavior, and motor abilities in adolescents and adults with Angelman syndrome.

The upcoming Phase 3 NEPTUNE trial, which expects to enroll approximately 60 children ages 4–12, will randomize the participants to take either oral OV101 or placebo, once daily for 12 weeks. The primary goal is to evaluate OV101’s effectiveness, measured by the change in CGI-I-AS scores, following treatment with OV101.

The CGI-I scale originally was developed by the National Institute of Mental Health (NIMH) to address clinicians’ views of the patients’ clinical state before and after initiating a study medication. Ovid optimized this scale to specifically evaluate Angelman symptoms, and called it CGI-I-AS.

Secondary goals (endpoints) will assess OV101’s benefit to improve sleep, communication, motor skills, socialization, daily living skills, and behavior.

The FDA granted OV101 orphan drug designation in September 2016 to treat Angelman syndrome

“Today, there are no approved medicines specifically for Angelman syndrome. Ovid previously received Orphan Drug and Fast Track designation for OV101 from the U.S. Food and Drug Administration (FDA). We recently announced we have initiated the pivotal Phase 3 NEPTUNE trial in Angelman syndrome and have a clear route to registration, if positive,” said Rakhit.