Current and emerging treatment options to prevent renal failure due to autosomal dominant polycystic kidney disease

Gopala K. Rangan*, Aarya Raghubanshi, Alissa Chaitarvornkit, Ashley N. Chandra, Robert Gardos, Alexandra Munt, Mark N. Read, Sayanthooran Saravanabavan, Jennifer Q. J. Zhang, Annette T. Y. Wong

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

4 Citations (Scopus)


Introduction: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common inherited cause of end-stage kidney disease (ESKD) in adults. The aim of this narrative review is to analyze current and emerging treatment options to delay ESKD due to ADPKD. Emerging treatments were defined as those that were in clinical trial (according to database to July 2020) or in development. Areas covered: The epidemiology and economic burden of ADPKD; molecular pathogenesis of ESKD; current (first-line; tolvaptan in groups with high-risk for progression to ESKD), emerging treatments under investigation [re-purposed small molecule drugs (SMDs): lixivaptan, venglustat, bardoxolone, tesevatinib, metformin; public health interventions: prescribed fluid intake, vitamin B3, ketone diet] and those in development (RGLS4326, VX-809, MR-L2, 2-doexyglucose). Expert opinion: Over the next decade, the number of proven treatments will expand, providing opportunities to individualize therapy based on personal preferences and disease ontology; Major barriers to future research include the absence of disease-specific biomarkers, national disease-specific registries. In parallel, there is also a need for need for earlier pre-symptomatic diagnosis and enhancement of health-care service delivery. Addressing these gaps will enable ESKD to become an ultra-rare complication of ADPKD during the 21st century.

Original languageEnglish
Pages (from-to)285-302
Number of pages18
JournalExpert Opinion on Orphan Drugs
Issue number8
Publication statusPublished - 2 Aug 2020
Externally publishedYes


  • Autosomal dominant polycystic kidney disease
  • kidney failure
  • treatment


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