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FDA Approval of Tolvaptan for PKD: Breakthrough Therapy vs. Value-Based Care

  • 1 Richard Lafayette, MD, is Editor-in-Chief of Kidney News. He is an investigator in the Otsuka PKD program and has received consulting fees in the past year.
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Richard Lafayette, MD

In April 2018, the U.S. Food and Drug Administration (FDA) approved the use of tolvaptan for the treatment of autosomal dominant polycystic kidney disease (PKD), allowing the United States to join most of the rest of the world in having the ability to deploy the first disease-specific therapy for PKD. Approval followed the release of results of the REPRISE trial, which demonstrated tolvaptan’s ability to meaningfully slow progression of stage 3 and 4 CKD over 12 months compared to placebo (1). The REPRISE study validated the results of the TEMPO trial, which initially provided strong

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