FDA Advisory Committee rejects tolvaptan for Polycystic Kidney Disease

The Cardiovascular and Renal Drugs Advisory Committee of the FDA has voted 9 to 6 not to approve Samsca (tolvaptan) for the treatment of autosomal dominant Polycystic Kidney Disease (ADPKD) which is a genetic illness characterised by the development of multiple cysts in the kidneys and which is the most common inherited kidney disease.

In a Phase III trial of tolvaptan in the ADPDK population, there were three patients with drug-induced liver injury which was attributed to the medicine. These patients did not progress to liver failure leading to transplantation or death but the adverse events nonetheless showed that tolvaptan has the potential to cause severe liver injury.