What is the role of tolvaptan in the treatment of autosomal dominant polycystic kidney disease (ADPKD)?

Updated: Mar 24, 2020
  • Author: Roser Torra, MD, PhD; Chief Editor: Vecihi Batuman, MD, FASN  more...
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Tolvaptan (Jynarque), a selective vasopressin V2-receptor antagonist, was approved in the United States in April 2018 to slow kidney function decline in adults at risk of rapidly progressing ADPKD. Approval was based on two phase 3 clinical trials.

Treatment with tolvaptan led to improvement in glomerular filtration rate (GFR) during the 1-year REPRISE (Replicating Evidence of Preserved Renal Function: an Investigation of Tolvaptan Safety and Efficacy in ADPKD) trial. The change from baseline in the estimated GFR (eGFR) was -2.34 mL/minute per 1.73 m2 in the tolvaptan group compared with -3.61 mL/min/1.73 m2 in the placebo group (P<0.001).<ref>37 </ref>In the extension trial (TEMPO 3:4) eGFR differences produced by the third year of the trial were maintained over the next 2 years of tolvaptan treatment.

The primary endpoint in the TEMPO 3:4 and TEMPO 4:4 studies was the intergroup difference for rate of change of total kidney volume (TKV). The trial met its prespecified primary endpoint of 3-year change in TKV (P<0.0001). The difference in TKV between treatment groups mostly developed within the first year, the earliest assessment, with little further difference in years 2 and 3. In years 4 and 5 during the TEMPO 3:4 extension trial, both groups received tolvaptan and the difference between the groups in TKV was not maintained. [38, 39]

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