FDA Approves New ADPKD Drug
The US Food and Drug Administration (FDA) has approved Jynarque (tolvaptan) for the treatment of adults with autosomal dominant polycystic kidney disease (ADPKD).
Jynarque is now the first FDA-approved drug treatment that can slow kidney function decline in adult patients with a high risk of rapidly progressing ADPKD.
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The approval was granted following the success of 2 trials: the 1-year REPRISE study and the 3-year TEMPO study. In the REPRISE study, treatment with tolvatapan resulted in a change in estimated glomerular filtration rate (eGFR) of -2.3 mL/min/1.73 m2/year from pretreatment baseline to post-treatment follow-up, compared with -3.6 mL/min/1.73 m2/year among those who received placebo.
During the TEMPO study, tolvaptan use was associated with a 1.0 mL /min /1.73m2 /year reduction in the rate of decline in eGFR compared with placebo among patients with earlier stages of ADPKD.
Serious and potentially fatal liver injury, acute liver failure requiring liver transplantation, and elevations of blood alanine and aspartate aminotransferases (ALT and AST) have been reported with the use of Jynarque.
To ensure patient safety, patients treated with Jynarque should undergo measurements of ALT, AST and bilirubin before initiating treatment, at 2 weeks and 4 weeks post-initiation, then monthly for 18 months and every 3 months thereafter, for the entire duration of treatment with Jynarque.
Due to these risks, Jynarque is only available through a restricted distribution program supported by an FDA-approved Risk Evaluation and Mitigation Strategy Program.
—Christina Vogt
Reference:
Otsuka’s JYNARQUE ™ (tolvaptan) approved by U.S. FDA as the first treatment to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD) [press release]. Tokyo, JP. Otsuka Pharmaceutical Co., Ltd. April 24, 2018. https://www.businesswire.com/news/home/20180424006422/en/Otsuka’s-JYNARQUE™-tolvaptan-Approved-U.S.-FDA-Treatment. Accessed on April 25, 2018.