The Food and Drug Administration has granted Orphan Drug Designation to an experimental therapy Marinus Pharmaceuticals is developing to treat Fragile X Syndrome.
That prompts two questions: What is Fragile X Syndrome, and how is the Radnor, Pennsylvania, biopharmaceutical company’s new drug candidate attempting to treat the disorder.
Fragile X syndrome is genetic condition that causes a range of developmental problems and symptoms, including cognitive impairment, learning disabilities and behavioral challenges. It is caused by a mutation in the FMR1 gene. Those with the condition exhibit autism-like symptoms including anxiety and mood swings, attention deficit and heightened reaction to stimuli.
The Centers for Disease Control and Prevention estimates about 100,000 people in the United States have Fragile X syndrome, with the condition affecting one in 3,600 to 4,000 males and one in 4,000 to 6,000 females of all races and ethnic groups. The CDC also estimates 7 percent of women and 18 percent of men with Fragile X syndrome have seizures.
Currently, there are no known cures or approved therapies for the disorder.
Marinus (NASDAQ: MRNS), founded in 2003, is developing ganaxolone in three different dose forms — IV, capsule and liquid — to treat the condition. Ganaxolone is designed to target a receptor in the central nervous system known as GABAA, a target known for anti-seizure and anti-anxiety activity.
Marinus previously studied ganaxolone as a potential treatment for seizures in adults, but halted that program after disappointed phase-III clinical trials results last June.
“We are pleased to receive Orphan Drug Designation from the FDA for ganaxolone in Fragile X Syndrome,” said Christopher M. Cashman, CEO of Marinus Pharmaceuticals, in a prepared statement. “This designation underscores the significant unmet medical need for children.”
Orphan Drug Designation is granted by the FDA Office of Orphan Products Development to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the United States. The designation provides the drug developer with a seven year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and a waiver of Prescription Drug User Fee Act filing fees.