Topline results of the Reprise Study in ADPKD have been announced and the good news continues for ADPKD. According to top-line results released by Otsuka, the maker of Tolvaptan:
- - Primary and key secondary endpoints were positive for tolvaptan vs. placebo in an additional Phase 3 clinical trial that examined the efficacy and safety of tolvaptan in autosomal dominant polycystic kidney disease (ADPKD)
- - The data are intended to address the Complete Response Letter (CRL) issued by the FDA for a New Drug Application (NDA) for tolvaptan in ADPKD in 2013
- - Trial results will be submitted for presentation at a nephrology medical congress in the second half of 2017
- The primary endpoint of the trial was the change in estimated glomerular filtration rate (eGFR) from pre-treatment baseline levels to post-treatment assessment. In patients treated with tolvaptan the reduction in eGFR was significantly less than in patients treated with placebo (p<0.0001). eGFR is an estimate of the sum of the filtration rates of all single functional nephrons (filtering units) in the kidneys, measured through creatinine-based estimation equations. The difference observed in this study represents a 35% reduction in the loss of kidney function compared to placebo in these patients over the course of one year. The key secondary endpoint was a comparison of the efficacy of tolvaptan treatment versus placebo in reducing the decline of annualized eGFR slope across all measured time points in the study. These data also showed significant benefit from tolvaptan vs. placebo (p<0.0001).
These results are preliminary until a peer-reveiwed publication is released, but at face-value, they add to the landmark findings of the TEMPO 3:4 study, reported previously on UKidney.