Marinus Pharmaceuticals in Radnor is getting a jumpstart over the competition in the race to develop a treatment for the rare, autism-like Fragile X Syndrome found in children.
The U.S. Food and Drug Administration’s recently awarded orphan drug designation gives Marinus the exclusive opportunity to market a product over the next seven years, along with access to tax, grant, and fee benefits, according to a Philadelphia Business Journal report by John George.
There are currently no other approved therapies for treating Fragile X.
“This designation underscores the significant unmet medical need for children,” CEO Christopher M. Cashman said.
Marinus’s new treatment is aiming to deliver ganaxolone – which targets a central nervous system receptor known for blocking seizures and anxiety – in IV, capsule, or liquid form.
Read more about the new Marinus breakthrough in the Philadelphia Business Journal here.
