Available Treatment

Out-of-pocket cost varies by insurance company and plan. Contact your insurer to find out whether tolvaptan is covered and what you would pay.

You need to be under the care of a nephrologist or experienced internist, and you must meet the criteria for being at risk of rapidly progressing ADPKD. Not every patient will qualify. For help finding an experienced nephrologist, visit the Find a PKD Clinic page.

Generally speaking, yes. It is the company’s intent to ensure all trial patients who want to continue taking the drug are able to do so. However, the drug will need to be prescribed by a nephrologist and previous study participants will undergo the same standard of care as all other patients receiving tolvaptan.

Every insurance company will negotiate to determine coverage for tolvaptan as a treatment for PKD and each individual plan available will be different. To find out if your insurance will cover tolvaptan, please reach out to your insurance company.

If you do not see a nephrologist regularly but you think you will benefit from tolvaptan, now is the time to set up an appointment. Take information about tolvaptan with you to your appointment and also, take a list of all of your questions. Keep in mind, not all patients will benefit from taking this drug and your nephrologist may tell you that you do not meet the criteria to be prescribed this drug. If this is the case, ask questions and be sure you understand why. For help finding a nephrologist with experience prescribing tolvaptan, visit our Find a PKD Clinic or connect with your local Chapter.

No. Tolvaptan is approved only for adults with ADPKD (18 and older), because it has not yet been clinically tested in children.

Tolvaptan has not been clinically tested in children at this time. Ongoing studies in Europe are evaluating the safety and efficacy in patients younger than 18.

We cannot comment on the interaction of specific drugs — this is a question you need to discuss with your nephrologist.

Yes. You will need regular blood tests because tolvaptan can cause liver function issues in some people. Starting tolvaptan and staying on it requires that you have a test to look at your liver function before you start, at week 2, at week 4, and then every month for 18 months. After that you must have a liver function blood test every 3 months. The tests measure two liver enzymes (ALT and AST) and bilirubin, a waste product filtered by the liver.

Tolvaptan is indicated to slow kidney function decline in adults at risk of rapidly progressing ADPKD. “Rapidly progressing” is not a defined standard – it takes many factors specific to each patient into account. Because of this, patients who are interested in the drug need to speak with their physician to consider all factors, as well as potential side effects, to decide if it is a good choice for them. If your risk of rapid progression is determined as high, it would be best to initiate the treatment early, even if kidney function is relatively normal. This will allow for maximum gain out of the tolvaptan therapy.

No. Tolvaptan is intended for patients with rapidly progressing ADPKD who have not had a transplant and are not on dialysis.

Tolvaptan is available in Japan, the European Union, Canada, the Republic of South Korea, Switzerland, Hong Kong, Australia, Turkey, and Taiwan. Approval in other countries will be subject to the regulatory agencies in each individual country.

Ongoing clinical studies have been done on humans to test the efficacy of water intake to slow disease progression in ADPKD. Until the results of these studies are available, it is not possible to compare the use of tolvaptan to that of water intake. However, ADPKD patients are generally advised to drink fluids throughout the day to suppress the thirst hormone (vasopressin). It is advised to discuss details of the water suppression with your nephrologist.

It isn’t possible to know exactly. In patients at risk of rapid progression, each four years of tolvaptan treatment is estimated to delay the need for dialysis or a kidney transplant by about one year.

Tolvaptan is approved for use in patient with rapid progressing ADPKD. Future studies are still needed to determine if tolvaptan could help reducing the liver cysts. At this time, tolvaptan is not indicated to slow progression of polycystic liver disease.

As far as we know, tolvaptan will not help or hurt cysts in the liver. The TEMPO trial showed no association between the severity of liver cysts/PLD and the risk for liver toxicity, therefore liver cysts/PLD are not associated with the liver toxicity issues associated with tolvaptan. You should consult your physician if you have concerns about liver cysts, PLD or any other liver disease and this drug.

The PKD Foundation not only supported early studies that led to the development of tolvaptan as a treatment but also helped guide PKD patients to the clinical trials.

“Today is a historic day in providing hope to patients with polycystic kidney disease, and we are thrilled to be a part of this first milestone to treat patients with ADPKD,” said Andy Betts, President and CEO of the PKD Foundation, when news of the approval was announced. “For the past 35 years, our goal has been to support PKD patients from care to cure. It is gratifying to play a part in the discovery of this treatment and to see it come to fruition.”

Betts added, “Many thanks to all of the patients who graciously took the time and resources to participate in the clinical trials to bring this treatment to the PKD community. This treatment would not exist without these patients.”

The time to develop a drug from inception to market is long, expensive and impossible to predict. There are several therapies in clinical trials now, but we can’t say how long it will take for the next one to be submitted for approval. Learn more about the latest in research and potential treatments here and explore clinical trials here.

The first study for ARPKD is underway now, and if the early results are positive, the study will continue to the next phase of clinical trials. To aid in the development of treatments for ARPKD, consider joining the ARPKD database. Visit arpkdb.org for more information or contact Research Coordinator Elena Gibson, RN with questions or to participate by calling 202-476-6877.

The PKD Foundation is committed to keeping our constituents informed about advances toward treatments for PKD. Subscribe to the PKD Blog and sign up to receive our emails. You can always learn about the latest in PKD research here.

Whether or not to have children screened for ADPKD is a very personal decision. The availability of tolvaptan as an approved treatment for ADPKD and the potential future therapies encourages individuals at risk of ADPKD (i.e. one of their parents have ADPKD) to consider screening. Screening children younger than 18 is not highly recommended unless a therapy is approved for patients younger than 18. Keep in mind that tolvaptan is approved for use in adults, 18 and over. Things to consider before screening children include:

• What will you do with the information once you have it?
• Will a diagnosis prevent you from obtaining medical or life insurance for your children? Are you better off knowing or not knowing?
• Can you financially afford the test (although ultrasound is relatively inexpensive, confirmation by genetic testing is expensive)?

Some people choose to remain undiagnosed but live a healthy lifestyle, eating well and monitoring blood pressure. They see the doctor often to monitor kidney function. If or when they have symptoms, they can revisit the decision to be tested. PKD is a progressive disease from year to year. Early diagnosis may allow the use of therapeutics earlier in the course of the disease and could also mean you are eligible to participate in clinical studies. All these factors and more must be considered before making a decision.