Breakthrough Therapy designation to HPBCD of Vtesse

News 1 February, 2016
Vtesse, Inc, a rare disease company, announced that FDA granted Breakthrough Therapy designation to VTS-270 (a proprietary form of HPBCD) for the treatment of children with Niemann-Pick Disease Type C (NPC).
The NPC Study, a Phase 2b/3 randomized, sham-controlled clinical trial designed to evaluate the efficacy and safety of VTS-270. Its primary objective is to evaluate the progression of the neurologic manifestations of NPC in children treated with VTS-270 compared to those who do not receive the drug.

“Breakthrough Therapy designation means that the FDA will provide intensive guidance and an organizational commitment to expedited development of VTS-270 for the treatment of NPC,” said Dr. Johnson. “These multidisciplinary interactions with the FDA will support Vtesse’s efficient design and conduct of its regulatory strategy with the goal of driving VTS-270 towards FDA approval.”!blank/uxqkj