Currently, there are no FDA-approved therapies for Niemann-Pick disease type-C1 (NPC). NPC is a rare lethal genetic lysosomal storage disorder that results in an accumulation of cholesterol in the liver and spleen and eventually leads to neurodegeneration. 2-hydroxypropyl-β-cyclodextrin (HPβCD) is a cyclodextrin typically used by the pharmaceutical industry as an excipient. Studies of NPC in animal models have shown that HPβCD can reduce the biochemical burden associated with NPC, improving neurological pathology, decreasing neurological dysfunction, and increasing lifespan. 

Niemann-Pick disease, type C (NPC) is a lethal, neurodegenerative disorder that affects children. Presently, no therapies for NPC are approved by the Food and Drug Administration (FDA). Several studies have suggested the potential use of 2-hydroxypropyl-β-cyclodextrin (HPBCD) to treat NPC, but the critical studies and data required for an Investigative New Drug (IND) application to evaluate HPBCD were not available.