Posted by Discovered in 1891, cyclodextrins, derived from starchy food, have found many applications in the cosmetic, agriculture, food and pharmaceutical industries as carriers of hydrophobic active substances due to biocompatible properties and their unique cage-like molecular structure that can form complexes with smaller molecules. Unexpectedly, recent research shows that cyclodextrins also can be active pharmaceutical ingredients such as artificial receptors and cholesterol modulators. Here, we discuss cyclodextrins with focus on their properties, their classical use as excipients, and their new applications as active ingredients in the following diseases: COVID-19, neuromuscular blockade, Stargardt, hemophilia, atherosclerosis, Alzheimer, Parkinson, diabetic kidney disease, dry eye disease, focal segmental glomerulosclerosis, Alport syndrome and Niemann–Pick C.
Interesting data: Sugammadex is already approved in 75 countries and marketed in more than 50 countries. In 2021, pharmaceutical sales of Bridion® exceeded US 1.5 billion (https://www.merck.com/news/merck-announces-fourth-quarter-and-full-year-2021-financial-results/)
Table 1 Development phase of HPBCD as drug
| Cyclodextrin | Drug name | Company | Disease | Development phase |
|---|---|---|---|---|
| Hydroxypropyl-β-cyclodextrin | Trappsol® Cyclo™ | Cyclo therapeutics | Niemann–Pick C | Phase 3 started in July 2021 |
| Alzheimer | Phase 2 started in September 2022 | |||
| – | Organo therapeutics | Parkinson | Jarazo et al. 2022 | |
| BRN-002 | Beren Therapeutics | Atherosclerosis | Preclinical trials in USA (IV) (2021) and phase I trial (In Volunteers) in New Zealand (IV) | |
| VAR200 | Zyversa Therapeutics | Focal segmental glomerulosclerosis (FSGS), Alport syndrome, diabetic kidney disease | Phase 2a study expected to begin in the first quarter of 2024 | |
| CBT-006 | Cloudbreak Therapeutics | Meibomian gland dysfunction (MGD) associated with dry eye disease (DID) | Phase 2 clinical trial accomplished in 2022 |
Kfoury, M., Lichtfouse, E. & Fourmentin, S. The revival of cyclodextrins as active pharmaceutical ingredients. Environ Chem Lett 23, 1–6 (2025). https://doi.org/10.1007/s10311-024-01782-8