DISCONTINUED: Stages of Development for Pioglitazone

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.

The master transcriptional regulator PGC1α, which controls mitochondrial biogenesis and antioxidant response was reduced in FA fibroblasts and in the KIKO mouse mode.

PPARγ agonists showed that the Azelaoyl-PAF increases frataxin mRNA and protein levels in fibroblasts derived from patients and pioglitazone restored anti-oxidant expression of SOD2 in FA fibroblasts and in the cerebellum of KIKO mice, through the activation of PGC1α.

2008: Dr. Rustin initiated a proof-of-concept trial in France to explore the effects of Pioglitazone on neurological function in FA patients. The trial recruited patients less than 22 years of age. Patients were treated for two years and underwent clinical exams and testing at six-month intervals during the study. Of note, individuals with Type I diabetes and those at risk for congestive heart failure should not take pioglitazone.

2013: The pioglitazone trial was completed but results have not been published.

This candidate has been removed from the pipeline chart because the study has been completed and it is unlikely there will be future studies of pioglitazone due its known side effects in individuals at risk for congestive heart failure.