Angelman Syndrome Treatment Granted Fast Track Designation by FDA

Mathew Shanley

Ovid Therapeutics announced yesterday that OV101 (gaboxadol), a potential therapy for Angelman syndrome, was granted Fast Track designation by the U.S. Food and Drug Administration (FDA). In September, the potential treatment option received orphan drug designation for the same indication.

“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 a press release.

Angelman syndrome is the result of a mutation in the UBE3A gene, which is found on chromosome 15 and the condition is characterized by developmental delay, lack of speech, seizures, and walking and balance issues. Patients with Angelman syndrome are highly susceptible to seizures and require lifelong care. At present, the neuro-genetic disorder occurs in approximately 1 in 16,000 live births, and there are no approved therapies for it.

OV101 has also previously received orphan drug designation for Fragile X syndrome, a common genetic form of intellectual disability and autism. It is a delta (δ)-selective GABAA receptor agonist, and targets the assumed underlying cause of Angelman syndrome, a disruption of the central physiological process of the brain, tonic inhibition.

“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.”

Ovid Therapeutics has reported that gaboxadol has been previously administered to more than 4,000 patients and was observed to have favorable safety and bioavailability profiles.

The first signs of Angelman syndrome typically present when a patient between 6 and 12 months of age, and seizures commonly begin between the ages of 2 and 3 years. Current standard-of-care includes medications to control seizure activity and other physical symptoms. Physical and occupational therapies, communication therapy, and behavioral therapies are vital in allowing individuals with the condition to reach their maximum development potential.

People with Angelman syndrome cannot be cured of the condition, however, they’re expected to live a normal life span.

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