Posted by Michael Wonder on 07 Oct 2023
Zatolmilast, an investigational treatment for Fragile X syndrome, receives rare paediatric disease designation from the US FDA
27 September 2023 - Shionogi announced the US FDA has granted Tetra Therapeutics, a Shionogi Group Company, rare paediatric disease designation for zatolmilast (BPN14770), an investigational treatment being studied for Fragile X syndrome, a leading cause of inherited intellectual disability and autism.
Zatolmilast is an investigational drug that is believed to work by modulating a signalling molecule called cyclic AMP (cAMP), which may promote the maturation of connections between neurons that are impaired in individuals with Fragile X syndrome.
