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.

For more information visit Vtessepharma.com.