Hope for people living with PKD: First-ever treatment approved by FDA

Imagine getting a diagnosis of a serious disease and then hearing your doctor say, "There is no treatment." That is what every adult with autosomal dominant polycystic kidney disease (ADPKD) heard — until now.

In some of the best news for the kidney community in a long time, the FDA recently approved a new drug that has been shown to slow down the progression of ADPKD to kidney failure.

Polycystic kidney disease (PKD) is a genetic disease that causes cysts to grow inside the kidneys. These cysts make the kidneys much larger than they should be and damage the tissue that the kidneys are made of, causing kidney disease, which can lead to kidney failure. PKD causes about 2% (2 out of every 100) of the cases of kidney failure in the United States each year.

ADPKD is the most common type of PKD. About 9 out of every 10 people with PKD have the autosomal dominant form, which is also the most common inherited kidney disease. ADPKD causes cysts to form only in the kidneys and symptoms of the disease may not appear until a person is between 30 and 50 years old.

The new drug, JYNARQUE™, is a pill taken twice a day that slows the growth of cysts and has been shown to preserve kidney function. It is a long-term use drug — extending the time before a person needs dialysis or a transplant, though it does not cure ADPKD. JYNARQUE™ was developed by Otsuka Pharmaceuticals.

Clinical trials were a critical part of developing this new medication. To learn more about other clinical trials for people living with kidney disease, please visit our clinical trials resource page.