BRIEF—Shionogi's zatolmilast gets FDA Rare Pediatric disease designation

The US Food and Drug Administration (FDA) has granted Tetra Therapeutics, now a part of Japanese drugmaker Shionogi, Rare Pediatric Disease designation for zatolmilast (BPN14770), an investigational treatment being studied for Fragile X syndrome (FXS), a leading cause of inherited intellectual disability and autism.

Zatolmilast was also awarded Orphan Drug designation by the FDA in 2018.

Shionogi acquired Tetra in 2020 as part of its mission to develop medications for unmet medical needs and thereby contribute meaningfully to society.

Tetra’s clinical programs, which focus on addressing cognitive function in FXS and other disorders, align strategically with Shionogi’s global vision.

Shionogi is advancing the zatolmilast development program with the intent, if it is approved, to offer people living with FXS the first cognitive treatment developed specifically for this rare genetic disorder.