FDA grants orphan designation to Fragile X syndrome therapeutic

The FDA has granted orphan drug designation to a small molecule therapeutic for individuals with Fragile X syndrome.

SPG601 is a novel calcium-activated potassium (BK) channel activator that works by binding to BK channels and increasing activation to restore synaptic function, according to a press release from Spinogenix Inc.

The FDA had cleared an investigational new drug application for SPG601 in April.

“We are thrilled that the FDA has recognized the potential of SPG601, marking an important milestone in our mission to address the significant unmet needs of those affected by Fragile X syndrome,” Spinogenix CEO Stella Sarraf, PhD, told Healio in an email.

“This designation not only underscores the critical demand for innovative treatments but also highlights our commitment to developing therapies that target the synaptic dysfunctions that we believe are at the heart of [Fragile X syndrome].”

In addition to intellectual disability, those with Fragile X may experience disabling symptoms such as severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression and developmental seizures. Many core symptoms of the condition have been linked to deficient activity of large conductance BK channels, according to the release.

Orphan drug designation is granted by the FDA to novel therapies developed to address rare diseases or conditions that affect fewer than 200,000 people in the United States. The status provides assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees, as well as 7 years of post-approval marketing exclusivity to drug manufacturers.