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

Spinogenix, Inc. has been granted Orphan Drug Designation by the U.S. FDA for SPG601, a novel treatment for Fragile X syndrome (FXS), highlighting the urgent need for new therapies. FXS, a leading cause of inherited intellectual disability and autism, lacks FDA-approved treatments. SPG601 aims to address core symptoms by modulating BK channels to restore synaptic function.

LOS ANGELES, Calif., May 20, 2024 — Spinogenix, Inc., a clinical-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SPG601 for the treatment of Fragile X syndrome (FXS). This designation underscores the critical need for innovative treatments for individuals with FXS, a condition for which there are currently no FDA-approved therapies.
FXS is the most common inherited form of intellectual disability and a known cause of autism, resulting from the silencing of the Fmr1 gene. It affects approximately 1 in 4-5000 men and 1 in 6-8000 women globally. Patients with FXS experience a range of debilitating symptoms, including severe anxiety, social aversion, hyperactivity, attention deficit, sensory hypersensitivity, aggression, and developmental seizures. Many of these symptoms are linked to deficient activity of large conductance BK channels.
SPG601 is a novel small molecule BK channel activator designed to bind to BK channels and increase their activation, thereby restoring synaptic function. This approach aims to address the core symptoms of FXS, potentially improving the lives of those affected by the condition.
Dr. Craig Erickson, Spinogenix Chief Medical Advisor, emphasized the significance of the ODD for SPG601, stating, "Receiving ODD for SPG601 underscores the high unmet need for new treatment options for individuals with FXS." Dr. Stella Sarraf, Spinogenix Chief Executive Officer, added, "We believe people living with Fragile X need a therapeutic option that works at the synaptic level to address their wide range of disabling symptoms."
The Orphan Drug Designation provides several benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees, and seven years of post-approval marketing exclusivity. Spinogenix is committed to advancing SPG601 through clinical trials as part of its mission to develop transformative therapeutics for conditions involving the loss or dysfunction of synapses.
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Reference News

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Spinogenix Announces U.S. FDA Orphan Drug ...
spinogenix.com · May 20, 2024

Spinogenix, Inc. received FDA Orphan Drug Designation for SPG601, targeting Fragile X syndrome (FXS), highlighting the u...

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