2-Hydroxypropyl-beta-cyclodextrin (HP-β-CD) (HP-β-CD)

2-Hydroxypropyl-beta-cyclodextrin (HP-β-CD) is a chemically modified cyclic oligosaccharide consisting of seven glucopyranose units (PubChem, CID 14126454). It is primarily utilized in the pharmaceutical industry as a functional excipient to enhance the solubility, stability, and bioavailability of hydrophobic drug molecules by forming non-covalent host-guest inclusion complexes (FDA, Inactive Ingredient Database). Beyond its role as an excipient, HP-β-CD has gained significant attention as a potential therapeutic agent for Niemann-Pick disease type C (NPC), a rare lysosomal storage disorder characterized by impaired cholesterol transport (NIH, NCATS). In this context, HP-β-CD facilitates the egress of accumulated unesterified cholesterol from lysosomes, effectively bypassing the defective NPC1 or NPC2 transport proteins (PubMed, PMID 20364142). It is also being explored for other conditions involving lipid dysregulation, such as atherosclerosis and Alzheimer's disease, due to its ability to modulate cellular lipid homeostasis (PubMed, PMID 27053774). However, its clinical use is associated with specific safety concerns, most notably dose-dependent ototoxicity leading to permanent sensorineural hearing loss (PubMed, PMID 28793970). Despite these challenges, it remains a critical tool in both drug formulation and the treatment of rare metabolic diseases.

Other names

HPBCDHydroxypropylbetadex(2-Hydroxypropyl)-beta-cyclodextrinHydroxypropyl-beta-cyclodextrin

Formation of host-guest inclusion complexes to sequester or mobilize hydrophobic molecules like cholesterol from cellular compartments.