FDA Approves Treatment for Kidney Function Decline

The FDA has approved tolvaptan (JYNARQUE) as the first drug treatment to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD), Otsuka Pharmaceuticals announced.

The approval is backed by data from the 2 phase 3 pivotal trials TEMPO 3:4 (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes) and REPRISE (Replicating Evidence of Preserved Renal Function: an Investigation of Tolvaptan Safety and Efficacy in ADPKD). The efficacy of JYNARQUE was demonstrated in the 2 pivotal trials that lasted 1 and 3 years, respectively. Data showed that JYNARQUE slowed kidney function decline in adults at risk of rapidly progressing ADPKD.

ADPKD is a progressively debilitating and usually painful genetic disorder that can lead to dialysis or kidney transplantation. It’s diagnosed in approximately 140,000 people in the US.

“The progression nature of ADPKD means that kidney function gets worse over time, eventually leading to end-stage renal disease. This progression happens more rapidly for some patients than others,” Michal Mrug, MD, associate professor, University of Alabama at Birmingham, investigator on REPRISE trial, said in a statement. “Today’s approval is great news for adults at risk of rapidly progressing ADPKD because by slowing the decline in kidney function, this therapy may give them more time before kidney transplant or dialysis.”

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