Vtesse, Inc., a rare disease company based in Gaithersburg, MD, announced that the U.S. FDA has granted drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), Breakthrough Therapy designation status. Both the FDA and the European Medicines Agency (EMA) had previously granted Orphan Drug status to VTS-270, which is currently in a pivotal Phase 2b/3 clinical trial. NPC is a progressive, irreversible, chronically debilitating – and ultimately lethal – genetic disease. It is caused by a defect in lipid transportation within the cell, which leads to excessive accumulation of lipids in the brain, liver and spleen. Researchers at the NIH’s National Center for Advancing Translational Sciences (NCATS) and Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), in close collaboration with Vtesse, patients and patient advocacy groups, developed VTS-270 as part of a project focused on finding treatments for NPC. VTS-270 has been shown to significant reduction of disease progression in animal studies and preliminary indications of efficacy in the Phase I study clinical trial.