FDA Grants Orphan Drug Status to Spinogenix's SPG601 for Fragile X Syndrome

Spinogenix, Inc., a biopharmaceutical company at the clinical stage, announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its drug candidate SPG601, intended for the treatment of Fragile X Syndrome (FXS). This designation highlights the significant need for new treatments for FXS, a condition currently lacking FDA-approved therapies.

Craig Erickson, M.D., Chief Medical Advisor at Spinogenix, emphasized the importance of this designation. He noted that individuals with FXS endure significant challenges, with no existing FDA-approved treatments. Spinogenix’s focus on FXS stems from the potential of SPG601 to address synaptic dysfunctions by modulating specific potassium channels, known as BK channels. This therapeutic approach aims to ameliorate core symptoms of FXS and substantially enhance the quality of life for affected individuals.

Fragile X Syndrome is recognized as the most common inherited form of intellectual disability and a leading cause of autism, linked to the silencing of the Fmr1 gene. The condition affects approximately 1 in 4,000 to 5,000 men and 1 in 6,000 to 8,000 women worldwide. Patients with FXS experience a range of debilitating symptoms such as severe anxiety, social withdrawal, hyperactivity, attention deficit, sensory hypersensitivity, aggression, and developmental seizures. Many of these symptoms have been associated with impaired activity of BK channels. SPG601 is a novel small molecule designed to activate BK channels, thereby restoring synaptic function.

Stella Sarraf, Ph.D., Spinogenix’s Chief Executive Officer, expressed enthusiasm regarding the ODD for SPG601. She highlighted the increasing recognition that synapse loss and dysfunction play crucial roles in a variety of diseases. According to Sarraf, there is a pressing need for therapeutic options that target synaptic level dysfunctions to address the wide array of symptoms experienced by individuals with FXS. She conveyed excitement over receiving this designation and the company's commitment to advancing small molecule therapies through clinical trials.

The FDA grants Orphan Drug Designation to investigational treatments targeting rare diseases affecting fewer than 200,000 people in the U.S. This designation provides developers with various benefits, including support in the drug development process, tax credits for clinical trial costs, exemptions from certain FDA fees, and seven years of post-approval marketing exclusivity.

Spinogenix is focused on developing groundbreaking therapeutics for conditions involving synapse loss or dysfunction. Their lead candidate is a synaptic regenerative therapy aimed at reversing synapse loss and enhancing cognitive and motor functions in neurodegenerative and neuropsychiatric disorders like ALS, Alzheimer’s disease, and schizophrenia. Additionally, the company is advancing a synaptic function therapeutic aimed at improving behavior in Fragile X Syndrome.