Crestor® (rosuvastatin)
Crestor inhibits HMG-CoA reductase, an enzyme found in the liver that plays a role in producing cholesterol. It is one of the drugs in the class known as statins that are used to lower cholesterol levels. Crestor was tested in FA to see if it would alter Apo-A levels, which were thought to be abnormal in FA.
INACTIVE: Stages of Development for Crestor
The drug development process can be thought of as a series of stages, and drugs must successfully pass through each stage to become available to patients. This treatment has been evaluated, and the program has been discontinued. Thus, it is not in the pipeline.
October 2018: Results from the open-label single-site pilot study of Rosuvastatin in 12 adults with FA were presented at the CHOP FA Symposium. The study revealed:
- Treatment with Rosuvastatin significantly lowered LDL levels but had modest effects on HDL, serum APOA1 levels and frataxin.
- Rosuvastatin altered fatty acid metabolism in platelets suggesting that the intervention did have a pharmacological effect on the pathway and confirming the role of HMG-CoA reductase in fatty acid metabolism.
- Rosuvastatin was well-tolerated in the majority of the subjects, but three subjects discontinued their participation early due to adverse events including elevations in creatinine kinase, liver function enzymes, and muscle cramping.
There is no data to suggest that treatment with rosuvastatin will impact other clinical features of FA. As this drug is approved and available for treatment of hypercholesterolemia, no further studies were initiated.