First Treatment for ADPKD is Approved

We’re excited to announce that on Tuesday, April 24, 2018, the U.S. Food and Drug Administration (FDA) granted approval of JYNARQUE™ (pronounced jin-AR-kew and also known as 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).

The treatment slows the growth of cysts and the progression of the disease and has been shown to preserve kidney function. The treatment for ADPKD is an oral pill taken twice a day. It is a long-term use drug – extending the time before a patient needs dialysis or a transplant, though not curing the disease. The drug was developed by Otsuka Pharmaceuticals.

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

“Today is an 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, CEO of the PKD Foundation. “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.”

Read the PKD Foundation’s official press release here

Questions about the new treatment? Call us at 1.800.PKD.CURE 


Frequently Asked Questions (Updated 4/28)

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. The wholesale cost for JYNARQUE ™ is $13,041.10/month.
The plan is for JYNARQUE ™ to be available by early June, although this is not guaranteed. It is a specialty drug and the current plan is for it to be available via mail-order from selected specialty pharmacies.
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 to receive the treatment and we will not know these criteria until the drug and its label are approved. For help finding a nephrologist, visit our clinics page at pkdcure.org/living-with-pkd/working-with-your-doctor/find-a-clinic/ or connect with your local Chapter, pkdcure.org/find-a-chapter/.
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.
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 JYNARQUE™.
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: pkdcure.org/what-is-pkd/latest-research/ Potential treatments information pkdcure.org/what-is-pkd/latest-research/pipeline Clinical trials information pkdcure.org/living-with-pkd/clinical-studies/ Research grants pkdcure.org/research-medical-professionals/research-funding/grants/
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 arpkdstudies.uab.edu for more information or contact Research Coordinator Elena Gibson, RN with questions or to participate by calling 202-476-2197.
The PKD Foundation is committed to keeping our constituents informed about advances toward treatments for PKD. Please visit pkdcure.org/email and sign up to receive emails to stay up-to-date and to learn about clinical trials in your area. Learn about the latest in PKD research: pkdcure.org/what-is-pkd/latest-research/pipeline/
Every insurance company will negotiate to determine coverage for JYNARQUE™ as a treatment for PKD and each individual plan available will be different. To find out if your insurance will cover JYNARQUE™, please reach out to your insurance company. Otsuka offers a number of support programs for eligible patients, including the JYNARQUE™ Copay Savings program, which ensures that eligible commercially insured patients do not pay more than $10 prescription for JYNARQUE™. Further information is available at www.JYNARQUE.com/Copay-Savings. The Otsuka Patient Assistance Foundation (OPAF), provides support for eligible patients who are unable to afford their medication. For more information, visit www.otsukapatientassistance.com.
If you do not see a nephrologist regularly but you think you will benefit from JYNARQUE™, now is the time to set up an appointment. Take information about JYNARQUE™ 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.
Whether or not to have children screened for ADPKD is a very personal decision. Keep in mind that JYNARQUE™ 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.
No, JYNARQUE™ is only approved for use in adult (18 and older) ADPKD patients.
JYNARQUE™ has not been clinically tested in children at this time.
We cannot comment on the interaction of specific drugs - this is a question you need to discuss with your nephrologist.
JYNARQUE™ will likely be issued from a specialty pharmacy. Every insurance company will have to negotiate and will determine how each plan available will cover this drug. As with any new prescription, patients will need to confirm coverage with your insurance company. Because of the risk of liver problems, JYNARQUE™ will only be available through a restricted distribution program called the JYNARQUE™ Risk Evaluation and Mitigation Strategy (REMS) Program. To ensure the safety of patients taking JYNARQUE™, 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 JYNARQUE™ (tolvaptan) treatment.
Yes. A REMS (risk evaluation and mitigation strategy) that patients and nephrologists will have to follow is described in the label. This will mean regular blood labs to monitor liver function and discussions with your physician.
JYNARQUE™ 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.
JYNARQUE™ is intended for patients with rapidly progressing ADPKD who have not had a transplant and are not on dialysis.
JYNARQUE™ is available in Japan, the European Union, Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey, Taiwan under the brand name JINARC. Approval in other countries will be subject to the regulatory agencies in each individual country.
No large clinical studies have been done on humans to test the efficacy of water intake to slow disease progression in ADPKD. As such, it is impossible to compare the use of JYNARQUE™ to that of water intake.
It is not possible to know exactly how long JYNARQUE™ may preserve kidney function. Clinical studies showed that the drug slowed the growth of cysts and preserved kidney function as compared to placebo.
JYNARQUE™ is approved for use in patient with rapid progressing ADPKD. This drug 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 JYNARQUE™. You should consult your physician if you have concerns about liver cysts, PLD or any other liver disease and this drug. Physicians can visit the JYNARQUE™ health care professional website at www.jynarquehcp.com for more information and contact information.