Autosomal-dominant polycystic
kidney disease (ADPKD)

Autosomal-dominant polycystic kidney disease (ADPKD) is the most common genetic kidney disease with an estimated prevalence of 1 in every 1000 to 2500 live births. ADPKD leads to progressive kidney function decline and in many patients finally to kidney failure with 70% of patients requiring renal replacement therapy in the form of dialysis or renal transplantation by the age of 70 years. The basic treatment includes strict blood pressure control and the use of blockers of the renin-angiotensin-aldosterone system. Tolvaptan,  a vasopressin V2 receptor antagonist, is the only specifically approved drug for the treatment of ADPKD, however coming with a number of significant and common side effects. There is a clear medical need for novel therapeutics for patients with ADPKD.