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

Spinogenix, Inc. has received Orphan Drug Designation from the FDA for its drug candidate SPG601, aimed at treating Fragile X Syndrome (FXS), a condition with no current FDA-approved therapies. SPG601 targets synaptic dysfunctions by modulating BK channels, offering hope for improving the quality of life for those affected by FXS.

Spinogenix, Inc., a clinical-stage biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its drug candidate SPG601 for the treatment of Fragile X Syndrome (FXS). This designation underscores the urgent need for new treatments for FXS, a condition that currently lacks FDA-approved therapies.
Craig Erickson, M.D., Chief Medical Advisor at Spinogenix, highlighted the significance of this designation, pointing out the severe challenges faced by individuals with FXS and the absence of FDA-approved treatments. SPG601's potential lies in its ability to address synaptic dysfunctions by modulating specific potassium channels, known as BK channels, aiming to alleviate core symptoms of FXS and significantly improve the quality of life for those affected.
Fragile X Syndrome is the most common inherited form of intellectual disability and a leading cause of autism, associated with the silencing of the Fmr1 gene. It affects approximately 1 in 4,000 to 5,000 men and 1 in 6,000 to 8,000 women globally. Symptoms include severe anxiety, social withdrawal, hyperactivity, attention deficit, sensory hypersensitivity, aggression, and developmental seizures, many of which are linked to impaired BK channel activity. SPG601, a novel small molecule, is designed to activate BK channels, thereby restoring synaptic function.
Stella Sarraf, Ph.D., Spinogenix’s Chief Executive Officer, expressed enthusiasm about the ODD for SPG601, emphasizing the critical role of synapse loss and dysfunction in various diseases. She stressed the need for therapeutic options targeting synaptic level dysfunctions to address the wide range of symptoms experienced by individuals with FXS. Sarraf also conveyed excitement over the designation and Spinogenix's dedication to advancing small molecule therapies through clinical trials.
The FDA's Orphan Drug Designation is granted to investigational treatments for rare diseases affecting fewer than 200,000 people in the U.S., offering benefits such as 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 committed to developing innovative 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. The company is also progressing a synaptic function therapeutic to improve behavior in Fragile X Syndrome.
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Reference News

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FDA Grants Orphan Drug Status to Spinogenix's SPG601 ...
synapse.patsnap.com · Jun 28, 2024

Spinogenix, Inc. received FDA Orphan Drug Designation for SPG601, targeting Fragile X Syndrome (FXS), a condition with n...

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