Nedosiran for primary hyperoxaluria

Nedosiran is currently in development for the treatment of primary hyperoxaluria (PH). PH is a group of rare genetic metabolic disorders that are characterized by the build-up of a substance known as oxalate in the kidneys and other organs of the body. This is due to low functional levels of a specific enzyme that normally prevents oxalate build-up. Excess oxalate binds to calcium that forms a hard compound (calcium oxalate) which can cause kidney and urinary stones. Recurrent stone formation and calcium oxalate in the kidney can cause chronic kidney disease (CKD) which can deteriorate kidney function and lead to kidney failure, and this can lead to oxalate build-up in other organs. The symptoms and severity of PH may vary greatly from one person to another. PH is an inherited disease. There is currently no cure for PH. Treatment focuses on protecting the kidneys to prevent kidney stones from developing, which includes drinking lots of liquid, low-oxalate diet and treatment with vitamin B6 as this can reduce oxalate build-up.