FDA grants fast-track designation to small molecule for Fragile X

The FDA has granted fast-track designation to an investigational small molecule to treat individuals with Fragile X syndrome, according to the manufacturer.

SPG601 is a small molecule, large-conductance, calcium-activated potassium (“BK”) channel activator that binds to BK channels and boosts activation, which in turn corrects specific synaptic dysfunctions that are a feature of core symptoms of Fragile X, Spinogenix Inc said in a press release.

“We are committed to helping people with Fragile X syndrome live better-quality lives, addressing the core symptoms by correcting specific synaptic dysfunctions,” Stella Sarraf, PhD, CEO and founder of Spinogenix, told Healio in an email. “The FDA fast-track designation will help us speed development of a much-needed therapeutic for Fragile X patients and their families, an underserved community who today have no approved treatments.”

In 2024, the FDA granted orphan drug designation to SPG601 for treatment of Fragile X. Fast-track designation will allow Spinogenix to expedite its development and review.

The company said in the release that topline results from a recently completed phase 2 clinical trial of the therapy are expected to be released by the end of the first quarter.