Ovid Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OV101 for the treatment of Angelman syndrome.

“This designation is an important milestone for both Ovid and the Angelman community as it enables increased dialog with the FDA, speeding our ability to bring this potential therapeutic option to people living with Angelman syndrome. We believe that OV101, with its novel mechanism of action, has the potential to be an innovative and impactful therapy,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics. “In addition to the regulatory milestones of orphan drug and Fast Track designations for Angelman syndrome, we achieved significant clinical progress with our OV101 program. As a result of positive Phase 1 data, we were recently able to expand our ongoing Phase 2 STARS clinical trial to include both adults and adolescents with Angelman syndrome. We look forward to data from the STARS trial in the second half of 2018.”

OV101 is a delta (δ)-selective GABAA receptor agonist that targets the disruption of tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of Angelman syndrome and other neurodevelopmental disorders. Ovid is currently studying OV101 in its Phase 2 STARS clinical trial, a randomized, double-blind, placebo-controlled study investigating the safety and efficacy of OV101 in patients with Angelman syndrome. Upon successful completion of a Phase 1 pharmacokinetic (PK) and safety study showing that OV101 has a similar PK profile in adolescents as in adults, Ovid recently amended the STARS protocol to include patients aged 13 years and older.

The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the FDA throughout the drug development and review process, often leading to faster drug approval and patient access.

About Angelman Syndrome
Angelman syndrome is a genetic disorder that is characterized by a variety of signs and symptoms. Characteristic features of this disorder include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders and anxiety. The most common cause of Angelman syndrome is the disruption of a gene that codes for ubiquitin protein ligase E3A (UBE3A). Angelman syndrome affects approximately 1 in 12,000 to 20,000 people in the U.S. There are currently no U.S. Food and Drug Administration (FDA)-approved therapies for the treatment of Angelman syndrome.
Angelman syndrome is associated with a reduction in tonic inhibition, a function of the delta (δ)-selective GABAA receptor that allows a human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. If tonic inhibition is reduced, the brain becomes inundated with signals and loses the ability to separate background noise from critical information.