Breakthrough Tx Status Granted to Drug for Debilitating Genetic Disease

First Genetic Identifier for Autism Found
First Genetic Identifier for Autism Found

Vtesse announced that the Food and Drug Administration (FDA) has granted VTS-270 Breakthrough Therapy designation for the treatment of Niemann-Pick Type C1 Disease (NPC), a progressive, irreversible, chronically debilitating, and ultimately lethal genetic disease.

NPC is caused by a defect in lipid transportation within the cell, leading to excessive lipid accumulation in the brain, liver and spleen.

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The FDA had previously granted Orphan Drug designation to VTS-270. It will be studied in a pivotal Phase 2b/3 clinical trial to assess its efficacy and safety for the treatment of NPC. The study is expected to enroll a total of 51 patients worldwide.

VTS-270 is a modified form of cyclodextrin being developed to target cholesterol storage in order to restore the cell's normal cholesterol metabolism and regulation.

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