In April 2018, the FDA granted Otsuka approval for tolvaptan (JYNARQUE) to treat autosomal dominant polycystic kidney disease (ADPKD). Jynarque is the first drug approved to slow the progression of kidney decline in ADPKD and marks the potential for a shift in the treatment paradigm. According to nephrologists the unmet need in PKD, even with Jynarque available, remains high and several late term agents are in clinical development including Genzyme’s venglustat and Reata’s bardoxolone methyl which is also being studied for a variety of glomerular diseases.
RealTime Dynamix™: Polycystic Kidney Disease (US) is a quarterly service designed to track the shifting landscape of PCK treatment. Each quarter, Spherix surveys 100 US based nephrologists who are actively managing patients with PKD and who meet other screening criteria (70% are office-based, must spend at least 50% of time in clinical practice, must be actively managing a minimum of 5 patients with ADPKD, in practice 2 to 40 years, agree to Spherix terms, regionally representative). Nephrologists complete a self-administered online survey of about 30 minutes in length with new content introduced each quarter. Nephrologists are limited to participating in this project twice per year.
This quarterly report has been fielded since Q1 2018.
Learn more about RealTime Dynamix™ reports here.
- How much of an unmet need exists in PKD relative to other conditions in dialysis and non-dialysis patients?
- What are the challenges of managing patients with PKD? How comfortable are nephrologists measuring total kidney volume?
- How are PKD patients characterized in terms of age, rate of CKD progression, co-morbid conditions, cyst development/presence, transplant eligibility, etc.?
- How often are inter-nephrology referrals taking place for patients with PKD?
- How is Jynarque being used in the treatment paradigm?
- What are the advantages and disadvantages of Jynarque?
- What percent of patients with ADPKD are candidates for Jynarque and what percent have actually been prescribed the drug?
- What are the barriers to the use of Jynarque? How are nephrologists navigating the REMS program, particularly for those in group practices?
- How much promotion is supporting Jynarque and what key messages are sales representatives delivering?
- What are the future near term projections for Jynarque?
- How familiar are nephrologists with PKD agents in development?
- What is their reaction to these products?
Bardoxolone methyl, venglustat, lixivaptan, tesevatinib