Ovid Therapeutics Inc (NASDAQ:OVID) announced on June 19 that its OV101 Angelman syndrome treatment received rare pediatric disease designation from the Food and Drug Administration (FDA).
The company has been developing OV101 as a delta (δ)-selective GABAA receptor agonist to combat a rare disease called Angelman syndrome. The announcement that the regulator awarded the treatment a rare pediatric disease designation marks an important milestone for the company and for patients that suffer from it.
Ovid Therapeutics is currently running a pivotal Phase 3 NEPTUNE trial to evaluate OV101 as a treatment for Angelman syndrome. The company is expected to disclose topline results from the study in Q4 2020. OV101 had previously garnered Fast Track designation, as well as Orphan Drug Designation from the FDA as an Angelman syndrome therapy.
The FDA awards Rare Pediatric Disease Designation to treatments that address rare but life-threatening illnesses that mainly affect children below 18 years old and which only less than 200,000 children suffer from in the U.S. If the FDA approves Ovid’s new drug application (NDA) for OV101, then the treatment will be put on priority review by the regulatory authority, and this may contribute to its fast-tracked availability in the market.
“OV101 has the potential to become the first FDA-approved therapy for individuals living with Angelman syndrome,” stated Ovid Therapeutics’ Chief Medical Officer, Dr. Amit Rakhit.
The rare pediatric disease designation provides further validation to Ovid’s work
Dr. Rakhit further pointed out that the recently awarded Rare Pediatric Disease Designation represents a great milestone for the company and for the program, thus highlighting the important work that they have been doing. He also added that the awarded designation for the treatment will make it eligible to receive a priority review voucher from the regulator. That would then provide more value to the work that the company has been doing, especially towards completing the pivotal Phase 3 NEPTUNE trial.
Dr. Rakhit also expressed gratitude to Congress and the FDA for passing the Creating Hope Act in 2012, to facilitate the Rare Pediatric Disease Designation. The company remains confident in the treatment’s ability to address the life-threatening disease.