The placebo-controlled, parallel-arm study demonstrated nearly 50% reduction in the change of total kidney volume among autosomal dominant polycystic kidney disease (ADPKD) patients as compared to placebo over a period of three years.
Mayo Clinic division of nephrology and hypertension professor of medicine Vicente Torres said ADPKD is the most common inherited and the fourth most common overall cause of kidney failure worldwide.
"In most patients with this disease, relentless cyst growth within the kidneys destroys the tissue, causes hypertension and painful complications, and negatively impacts the quality of life," Torres added.
"The results of this study reveal a potential treatment that blunts kidney growth, lessens associated symptoms and slows kidney function decline when given over three years."
A significant reduction in the risk of a composite events of worsening kidney function, kidney pain, hypertension or albuminuria were observed, meeting the key secondary endpoint of the randomized, double-blind study.
The common adverse events associated with tolvaptan therapy were linked to its aquaretic mode of action while common adverse events reported in placebo included renal pain, haematuria and urinary tract infection.