Allen Davidoff, PhD, CEO of XORTX Therapeutics, gives an overview of autosomal dominant polycystic kidney disease (ADPKD).
As Dr. Davidoff explains, ADPKD is a genetic disorder characterized by the formation of cysts in the kidneys. The most common symptoms – hypertension, flank pain, hematuria, and kidney insufficiency – are caused by cyst formation. In most patients, ADPKD leads to end-stage renal disease which requires either dialysis or a kidney transplant. ADPKD is caused by mutations of one of two genes responsible for proper function of the kidneys and other parts of the body. Approximately 85% have ADPKD1, the most aggressive form of the disease.
Currently, there is one therapy approved by the U.S. Food and Drug Administration (FDA) for ADPKD, tolvaptan. However, due to its significant risk of side effects, it is generally limited to those most severely affected by ADPKD. This leaves approximately 90% of patients with ADPKD without a targeted therapeutic option.
Oxypurinol, an orphan drug being developed by XORTX Therapeutics, is currently under investigation as a treatment for patients with stage 2-4 ADPKD. The first of two phase 3 studies is planned to start in the second half of 2023. To learn more about the clinical trials and how to register, visit https://www.xortx.com/
To learn more about ADPKD and other rare kidney disorders, visit https://checkrare.com/diseases/kidney-and-urinary-diseases/
