Polycystic Kidney Disease – Special Topics – Special Topic: Assessment & Forecast – US, EU5, Japan, China

Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by the development and progressive enlargement of fluid-filled cysts in the kidneys. Particularly in the early stages, the disease is often asymptomatic and detectable only through diagnostic imaging of the kidney to reveal cysts. The launch of Otsuka’s Jynarque / Jinarc / Samsca (tolvaptan) introduced the first disease-specific therapy for ADPKD. However, several therapies are being investigated in clinical trials, including Sanofi / Genzyme’s venglustat, Reata Pharmaceuticals’ bardoxolone methyl, Palladio Bioscience’s lixivaptan, and Regulus Therapeutics’ RGLS-4326. These therapies are forecast to fulfill several of the unmet needs that remain. These unmet needs include superior efficacy and safety / tolerability compared to tolvaptan, and prescribing to a greater range of patients (e.g., those with elevated liver enzymes).


  • What do key opinion leaders think about the emerging therapies Sanofi / Genzyme’s venglustat, Reata Pharmaceuticals’ bardoxolone methyl, Palladio Bioscience’s lixivaptan, Regulus Therapeutics’ RGLS-4326, XORTX Therapeutics’ oxipurinol, and Galapagos’ GLPG2737? What is the likelihood that these drugs will launch? How will emerging therapies differentiate themselves in an increasingly crowded market?
  • How will the anticipated genericization of Otsuka’s Jynarque / Jinarc / Samsca affect the uptake of the branded and emerging therapies?
  • What are the different reimbursement challenges that ADPKD therapies face in the United States, Europe, Japan, and China?
  • What are the key unmet needs in ADPKD, and how likely are they to be met?


  • United States, EU5, Japan, China

Primary research:

  • 20 country-specific interviews with thought-leading nephrologists
  • Supported by survey data collected for this and other DRG research


  • Diagnosed, and drug-treated prevalent cases of ADPKD by country.


  • 15-year, annualized, drug-level sales and patient share of key ADPKD therapies through 2020, segmented by brands / generics and epidemiological subpopulations.

Emerging therapies

  • Phase III/PR: 3 drugs; Phase I/II: 3 drugs


Special Topic: Market Assessment & Forecast provides market intelligence with world-class epidemiology, insight into current and future treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.

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