Ovid Therapeutics (NASDAQ:OVID) reported that, following a positive opinion from the European Medicines Agency, the European Commission granted OV101 orphan drug designation for the treatment of Angelman syndrome.
Angelman syndrome is a rare genetic disorder that affects the brain’s ability to correctly process excitatory and inhibitory neurological signals, a function of a specific GABA receptor. OV101, or gaboxadol, is a selective agonist of that receptor.
Patients with Angelman syndrome experience delayed development, intellectual disability, speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. There are currently no approved therapies for the condition.
“OV101 is the only medicine currently in clinical development for Angelman syndrome,” Dr. Amit Rakhit, Ovid’s CMO and head of research and development, said in a statement.
“It’s our intent for OV101 to be the first medicine to be approved for this important and underserved community,” he added.
Ovid recently initiated a Phase 3 trial of OV101 in Angelman syndrome patients aged four to 12 years old, and expects topline results in mid-2020.