adrabetadex

Adrabetadex (VTS-270) is an investigational small molecule drug consisting of a specific mixture of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD). It was developed for the treatment of Niemann-Pick disease type C1 (NPC1), a rare and fatal lysosomal storage disorder caused by mutations in the NPC1 or NPC2 genes, which lead to the toxic accumulation of unesterified cholesterol and glycosphingolipids in the endolysosomal system. Adrabetadex acts by facilitating the mobilization and egress of sequestered cholesterol from lysosomes, thereby reducing lipid storage and potentially slowing the progression of neurological and visceral manifestations. The drug was developed through a collaboration between the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Vtesse (later acquired by Mallinckrodt), and Janssen Pharmaceuticals. Clinical development included both intravenous administration for liver disease and intrathecal administration to bypass the blood-brain barrier for neurological symptoms. However, the Phase 2b/3 clinical program was terminated in early 2021 after data failed to demonstrate a clear clinical benefit over the risks.

Brand names

Trappsol Cyclo

Other names

2-hydroxypropyl-beta-cyclodextrinHP-beta-CDHPBCDhydroxypropyl-beta-cyclodextrin