blue U.S. Food & Drug Administration (FDA)
green European Medicines Agency (EMA)
PKD is marked by a long period of stable kidney function (as measured by the currently accepted endpoints in drug development) during which the kidneys expand enormously due to cyst growth. Serum creatinine levels, the currently accepted endpoint, only rise once the kidneys have incurred serious, irreversible damage. Without a better endpoint, the development of therapeutics to slow or stop cyst growth at an early stage of the disease is impeded. The PKD Outcomes Consortium is developing evidence to support the use of imaging of total kidney volume (TKV) as a prognostic biomarker of the rate of disease progression to select patients likely to respond to therapy into clinical trials.