In September of 2011 the Mayo Clinic partnered with IC-MedTech to investigate the use of Apatone to treat polycystic kidney and liver diseases (PKD/PLD). These genetic, high-morbidity diseases are characterized by uncontrolled growth of cysts that eventually compromise basic organ functions and, untreated, may lead to organ failure. PKD/PLD afflict an estimated 600,000 people in the U.S. and 2.5 million worldwide. These kidney and liver cystogenic diseases are incurable and refractory to most currently available therapies. Advanced stages usually require dialysis and/or organ transplant giving rise to significant unmet medical needs.
Based on the results from collaborative efforts between Mayo Clinic and IC-MedTech, in April 2013, Apatone became the first investigational drug ever granted orphan designation by the FDA for both polycystic kidney disease and polycystic liver disease.