Ovid Therapeutics’ (NASDAQ:OVID) OV101 received FDA rare pediatric disease 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.
OV101 is currently being evaluated for the treatment of Angelman syndrome in a Phase 3 trial, with topline results expected in the fourth quarter of 2020.
“OV101 has the potential to become the first FDA-approved therapy for individuals living with Angelman syndrome,” Dr. Amit Rakhit, Ovid’s president and CMO, said in a statement.
“Receiving rare pediatric disease designation from the FDA is a significant milestone for this program and underscores the critical value of our work. Importantly, with this designation, we may be eligible to receive a priority review voucher from the FDA, providing significant value as we work diligently towards the completion of our ongoing pivotal Phase 3 NEPTUNE trial,” he added.
OV101 has previously been granted FDA orphan drug and fast track designations, and orphan drug designation in Europe.
