Lumasiran is in clinical development for the treatment of primary hyperoxaluria type I (PH1). PH1 is a very rare disease caused by certain genetic mutations, in which excess oxalate production results in the deposition of oxalate crystals in the kidneys and urinary tract. This leads to stone formation and kidney failure with significant morbidity and mortality. Treatment options for PH1 include vitamin B6 which is known to reduce the body’s production of oxalate, dietary recommendations to prevent kidney stones and combined liver-kidney transplantation before or after development of end-stage kidney failure.
Lumasiran, which is administered as a subcutaneous injection, is designed to reduce the levels of an enzyme called glycolate oxidase produced by the liver. Oxalate production is therefore inhibited. By reducing oxalate production, lumasiran has the potential to prevent the actual disease process that develops in PH1. If licensed, lumasiran may provide the first pharmacological treatment option for patients with PH1 who do not have any approved treatment.
Vadadustat is in development for the treatment of anaemia in patients with chronic kidney disease (CKD). Anaemia is a condition where the body has fewer red blood cells (RBC) to carry oxygen throughout the body resulting in a decline in function of the body’s organs and tissues. Symptoms of anaemia in CKD patients include weakness, fatigue, headaches, dizziness and difficulty breathing. Anaemia is common in people with CKD because diseased kidneys produce less erythropoietin (EPO) which is needed to make RBCs. Current treatment for anaemia in CKD involves erythropoietin stimulating agents (ESAs) however, they may result in adverse side-effects so there is a need for safer treatment options.