Tolvaptan treatment for ADPKD

Early in the disease, there are generally no symptoms at all. In fact, many people are never diagnosed with PKD because they have few or no symptoms. Often the first sign of PKD is high blood pressure, blood in the urine or a feeling of heaviness or pain in the back or abdomen. Sometimes the first sign may be a urinary tract infection or kidney stones.

On April 24, 2018, the U.S. Food and Drug Administration (FDA) granted approval of tolvaptan to be the first treatment in the United States for adult patients with autosomal dominant polycystic kidney disease (ADPKD), the most common form of polycystic kidney disease (PKD).

Tolvaptan is a medication (taken twice a day as an oral pill) that affects how the kidneys control the concentration of urine. It has been shown to slow down the growth of kidney cysts (total kidney volume) when it is taken for a long time (several years) by adults at risk of rapidly progressing ADPKD. This may help protect the function of your kidneys and delay the need for a kidney transplant or dialysis. Your kidney function would continue to decline, but at a slower rate.

Known major side effects:

Thirst and frequent urination are the most common side effects. Making large amounts of urine and urinating at night are also common. Rare but potentially serious liver injury is possible. Patients must be under the care of the prescribing physician to monitor all side effects and the drug should be discontinued at the first sign of liver effects. This effect is most often reversible when the drug is stopped based on the first finding of elevated liver enzymes in a blood test.

Watch our webinar to learn about the first treatment for ADPKD. Hosted by Ronald Perrone, M.D.

Frequently Asked Questions

How much will it cost?

The out of pocket expense for each patient will vary depending on their insurance company and the type of plan chosen. Insurance companies will negotiate to determine the price they pay for the drug and will determine what consumers pay based on their coverage. 

How do I obtain the treatment?

Patients will need to be under the care of a nephrologist or experienced internist who has read the latest study results and understands the requirements and steps necessary to prescribe this drug. Please keep in mind that this drug will not be prescribed for all ADPKD patients. Patients will have to meet the criteria of at risk for rapidly progressing ADPKD based on results from the clinical trials. For help finding a nephrologist with experience prescribing tolvaptan, visit our Find a PKD Clinic page or connect with your local Chapter.

Will those already on tolvaptan through clinical trials be able to continue treatment?

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.

Will my health insurance cover 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. 

How do I find a physician that will prescribe tolvaptan?

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 page or connect with your local Chapter.

Can children take tolvaptan?

No, tolvaptan is only approved for use in adult (18 and older) ADPKD patients.

Why can't this treatment be given to children under the age of 18?

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.

Can I take tolvaptan if I am on other medications (BP, reflux, etc.)?

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

Will patients receiving tolvaptan be required to get labs?

Yes. A REMS (risk evaluation and mitigation strategy) that patients and nephrologists will have to follow is described in the label. To ensure the safety of patients taking tolvaptan, it is necessary to measure ALT, AST and bilirubin before initiating treatment, at 2 weeks and 4 weeks after initiation, then monthly for 18 months and every 3 months thereafter, for as long as the patient is on tolvaptan treatment. This will mean regular blood labs to monitor liver function and discussions with your physician.

At what point in progression is the best time to start taking tolvaptan?

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 it relatively normal. This will allow for maximum gain out of the tolvaptan therapy.

Would this treatment benefit a patient that has had a transplant but still has native kidneys?

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

Is tolvaptan available in other countries outside of the United States?

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.

Would increased water intake slow down progression of PKD as a substitute for taking tolvaptan?

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.

How long will tolvaptan help preserve kidney function?

It is not possible to know exactly how long tolvaptan may preserve kidney function. Clinical studies showed that the drug slowed the growth of cysts and preserved kidney function as compared to placebo. In patients with risk of rapid progression, it is estimated that each four years of tolvaptan treatment could delay the need for renal replacement therapy (dialysis or transplant) by one year.

Will tolvaptan help slow progression of polycystic liver disease (PLD)?

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. 

How was the PKD Foundation involved in efforts to get tolvaptan approved as a treatment for ADPKD?

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.”

When should the next treatment be on the market?

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

What about a treatment for ARPKD? Is anything happening for these patients?

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 for more information or contact Research Coordinator Elena Gibson, RN with questions or to participate by calling 202-476-6877.

How can I stay updated on the latest in PKD research?

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.

Should I get my children screened for ADPKD?

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.

Content last updated November 2, 2020 — Reviewed by Fouad Chebib, M.D. (Mayo Clinic)

Page last reviewed November 2022