Fragile X Treatment Gets Fast Track Designation
Post Date: January 14, 2025 | Publish Date:
Cincinnati Children’s expert served as principal investigator of recently completed Phase 2 clinical trial
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SPG601, a treatment for people with Fragile X syndrome (FXS) made by the Los Angeles-based company Spinogenix.
The treatment helps restore synapse function for people with FXS, the leading inherited form of intellectual disability and a known cause of autism that results from the silencing of the Fmr1 gene. FXS affects approximately 1 in 4-5000 men and 1 in 6-8000 women globally. In addition to intellectual disability, FXS symptoms can include severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, and developmental seizures.
Craig Erickson, MD, Division of Child and Adolescent Psychiatry at Cincinnati Children’s, has been studying FXS for years. He was the principal investigator of a recently completed Phase 2 study conducted at Cincinnati Children’s to evaluate the treatment. Erickson also serves as Chief Medical Advisor for Spinogenix.
“I am thrilled that the FDA has granted Fast Track designation to SPG601 for FXS, highlighting its potential as a novel therapeutic option for a disease that, to this day, has no approved treatment,” says Craig Erickson, MD, in a statement released by Spinogenix. “The possibility that SPG601 could address underlying synaptic deficits central to this condition in people with FXS brings immense hope, and with this designation, we are even closer to making it available to patients sooner.”
The FDA granted Orphan Drug Designation to SPG601 in May 2024.
Read more about the treatment and its fast track status
