FDA grants orphan drug status to ganaxolone for Fragile X syndrome

The FDA has granted orphan drug designation to ganaxolone for the treatment of Fragile X syndrome, the most common genetic cause of autism, according to a press release from Marinus Pharmaceuticals.

“This designation underscores the significant unmet medical need for children suffering from a genetic mutation that causes autism-like symptoms including anxiety, mood swings and attention-deficit,” Christopher M. Cashman, president and CEO of Marinus, said in the release.

Fragile X syndrome is caused by an FMR1 gene mutation and can be diagnosed by several cognitive, behavioral and educational impairments. About 100,000 people in the U.S. have Fragile X syndrome, which affects 1 in 3,600 to 4,000 men and 1 in 4,000 to 6,000 women of every race and ethnicity, per the CDC.

Seizures are the most recognizable feature of Fragile X syndrome; which about 7% of women and 18% of men experience. However, there are currently no known cures or approved therapies for the syndrome.

Ganaxolone is an endogenous neurosteroid produced in the central nervous system that acts on the synaptic and extrasynaptic GABAA target to suppress seizures and anxious activity.

Marinus — which plans to develop ganaxolone into IV, capsule and liquid forms — has conducted clinical trials over 2 years to evaluate the safety and efficacy of ganaxolone as adjunctive therapy for seizures and anxiety in Fragile X syndrome. Study outcomes indicated that ganaxolone is safe and well-tolerated overall, however, common reported adverse events were somnolence, dizziness and fatigue.

The FDA’s Office of Orphan Products Development assigns orphan drug designation to novel drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect less than 200,000 people nationwide.

Disclosures: Cashman is employed by Marinus Pharmaceuticals.