Saturday, June 20, 2020
Ovid Therapeutics Inc., a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to OV101 (gaboxadol) for the treatment of Angelman syndrome. OV101 is believed to be the only delta (δ)-selective GABAA receptor agonist in development and is currently being evaluated in the Company’s pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020. The FDA has previously granted Orphan Drug and Fast Track designations for OV101 for the treatment of Angelman syndrome.
Under the Creating Hope Act passed into federal law in 2012, the FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the U.S. If a new drug application (NDA) for OV101 in Angelman syndrome is approved, Ovid may be eligible to receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application or may be transferred and/or sold to other companies for their programs, such as has recently been done by other voucher recipients.
“OV101 has the potential to become the first FDA-approved therapy for individuals living with Angelman syndrome. Receiving Rare Pediatric Disease Designation from the FDA is a significant milestone for this program and underscores the critical value of our work,” said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of Ovid Therapeutics. “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. We are grateful to the FDA and Congress for having enacted this law which helps Ovid and all companies developing innovative drugs for rare pediatric conditions.”