Fragile X Syndrome Gets FDA Fast Track Designation

Ovid Therapeutics, Inc. announced this morning that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to gaboxadol (OV101), a therapy in development for the treatment of Fragile X syndrome.

Gaboxadol, a delta (δ)-selective GABA_A receptor agonist, is the first investigational drug being developed to target what is believed to be the underlying cause of Fragile X syndrome and other neurodevelopmental disorders: a mutation in the FMR1 gene.

The genetic mutation can cause the disruption of toxic inhibition, a central physiological process of the brain, and can result in autism-like symptoms such as cognitive impairment, anxiety, mood swings, hyperactivity, attention deficit, poor sleep, self-injury and heightened sensitivity to various stimuli, like sound.

According to the National Institute of Health (NIH), Fragile X syndrome is the most common genetic cause of autism, and affects 1 in approximately 3,800 males and 1 in 5,000 females. Because the genetic mutation is X-linked, males are susceptible to more severe symptoms.

“Fragile X syndrome continues to have a high unmet medical need and has no FDA-approved therapies available. Fast Track designation enables us to work closely with the FDA to accelerate our efforts to potentially provide an impactful therapy to people with Fragile X syndrome,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics in a press release. “We are committed to exploring the potential of OV101 to become a transformative medicine and we look forward to announcing further progress this year.”

Ovid recently completed a successful Phase 1 study evaluating the pharmacokinetics (PK) and safety of gaboxadol in patients with Fragile X syndrome and Angelman syndrome.

The Phase 1 single, dose, single-arm, open-label clinical trial enrolled 7 male and 5 female patients, aged 13 to 17, who had been diagnosed with either Angelman syndrome or Fragile X syndrome. All participants receieved a single 5mg oral dose of OV101, and overall results of the PK study met the objectives, showing that PK parameters in adolescents with these conditions were not significantly different from previous data generated in young adults.

The data collected will advise dosing in future clinical trials, and backs the advancement of the development of gaboxadol for the treatment of adolescents with Fragile X syndrome.

The company plans to initiate the Phase 2 STARS trial in 2018, investigating the safety and efficact of OV101 in males ages 13 to 22 years who have been diagnosed with Fragile X syndrome. The trial is expected to enroll approximately 75 patients, and has several exploratory endpoints to evaluate measures of gross and fine motor skills, maladaptive behavior, sleep, clinical global impression and health-related quality of life (QOL) questionnaires.

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