Vtesse Granted Breakthrough Therapy Designation for Neimann-Pick

January 25, 2016

Vtesse, Inc., which launched in January 2015, is the first spin-off company for orphan drug accelerator, Cydan Development, Inc who today announced that the FDA has granted a breakthrough therapy designation to its lead drug candidate, VTS-270 for treatment of Niemann-Pick Type C1 Disease (NPC), an ultimately lethal genetic disease.

VTS-270 has the potential to be the first effective treatment for slowing or stabilizing disease progression in children and adolescents. This designation is supported by strong preclinical and early clinical data with VTS-270, including that from the Phase 1 study conducted by the National Institutes of Health(NIH).

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.

Vtesse expects to enroll a total of 51 patients at up to 20 sites (across the United States, the European Union, and other countries) to participate in its pivotal Phase 2b/3 clinical trial of VTS-270 for treatment of NPC.

For more information on Vtesse’s pivotal Phase 2b/3 clinical trial, visit



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