One develops anaemia when their circulating level of red blood cells are low, meaning inadequate oxygen is delivered to the various tissues in the body. Anaemia is caused by multiple factors. However, the shortage of iron is the most common. Anaemia of chronic kidney disease (CKD) is caused either by low levels of a hormone principally produced in the kidneys called erythropoietin (EPO for short), or by the disease process causing less iron from the gut to be available for use by the body. EPO, when released by the kidney, stimulates the bone marrow in the body to produce red blood cells. In CKD, low levels of EPO cause poor functioning of this pathway, often thereby causing anaemia. Possible adverse effects of anaemia include fatigue, increased cardiac output, heart failure, reduced cognition and concentration, reduced libido and reduced immune responsiveness.
Roxadustat is a product in development that can be taken orally as a tablet. It has the potential to stimulate the growth of red blood cells by increasing the body’s own production of EPO in patients with CKD who may be on dialysis or pre‐dialysis. If approved for the treatment of anaemia in this population, roxadustat will provide an alternative treatment option that offers a likely reduction in the need for supplemental iron as an advantage.
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.