Pharma Focus America

Spinogenix Receives FDA Orphan Drug Designation for SPG601 to Treat Fragile X Syndrome

Tuesday, May 21, 2024

Spinogenix, Inc., a clinical-stage biopharmaceutical company focused on developing innovative therapies to restore synapses and improve patients' lives, has announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SPG601 for treating Fragile X Syndrome (FXS).

Dr. Craig Erickson, Spinogenix's Chief Medical Advisor, emphasized the significance of receiving ODD for SPG601, indicating the urgent need for new treatment options for individuals with FXS. Currently, there are no FDA-approved therapies for FXS. Spinogenix's decision to concentrate on FXS is based on SPG601's potential to address specific synaptic dysfunctions by modulating calcium-activated potassium ("BK") channels, which are implicated in FXS symptoms.

FXS, the most common inherited form of intellectual disability and a known cause of autism, results from the silencing of the Fmr1 gene. It is considered an orphan disease, affecting approximately 1 in 4-5000 men and 1 in 6-8000 women globally. FXS patients suffer from various disabling symptoms, including severe anxiety, social aversion, hyperactivity, attention deficit, sensory hypersensitivity, aggression, developmental seizures, among others. Many of these symptoms are linked to deficient activity of large conductance BK channels. SPG601, a novel small molecule BK channel activator, aims to bind to BK channels and increase their activation to restore synaptic function.

Spinogenix, expressed enthusiasm about the ODD designation for SPG601, underscoring the company's commitment to advancing small molecule therapies to address the diverse range of disabling symptoms experienced by individuals with Fragile X.

Orphan Drug Designation, granted by the FDA to investigational therapies targeting rare medical conditions affecting fewer than 200,000 people in the U.S., provides several benefits to drug developers, including assistance in the drug development process, tax credits for clinical expenses, exemptions from certain FDA fees, and seven years of marketing exclusivity post-approval.



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