KANSAS CITY, Mo., April 24, 2018 /PRNewswire/ -- The PKD Foundation is excited to announce that on April 24 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 rapidly progressing autosomal dominant polycystic kidney disease (ADPKD), the most common form of polycystic kidney disease (PKD). 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 JYNARQUE™ clinical trials. JYNARQUE™ was developed by Otsuka Pharmaceuticals.
PKD, a chronic, genetic disease, is characterized by uncontrolled growth of cysts in the kidneys and other organs and can lead to kidney failure. Before today, there has been no treatment specifically for PKD in the U.S. The only treatment options were to receive a transplant or begin dialysis. And though there is no cure, patients who are prescribed JYNARQUE™ for PKD will see a slower decline in their kidney function, leading to improved health and well being.
For Nicole Harr, this means there is hope for living a longer, fuller life with PKD.
"It is very difficult to live with a chronic disease that has no treatment and no cure that progresses over time," Harr said. Both her dad and grandfather passed away from PKD. She has two children, and the fear that they could have PKD is in the back of her mind, she said.
For almost 10 years, Harr participated in a clinical trial that tested JYNARQUE™ and says she did it for her children. With today's news, she is "grateful for the opportunity to contribute to research that drove us to a treatment. My hope is for my children and for future PKD patients."
And hope for patients like Harr, her children and future generations of PKD patients is exactly what the PKD Foundation has tirelessly worked toward for more than 35 years.
"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."
Following this approval by the FDA, JYNARQUE™ will be available in the United States for patients in the coming weeks.
JYNARQUE™ 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 efficacy of JYNARQUE™ in ADPKD was based on data from two of the largest Phase 3 clinical trials in patients with ADPKD. Lasting one and three years respectively, they led to JINARC®/JYNARQUE™ to be approved for adult patients with ADPKD in Japan, the European Union, Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey, Taiwan and finally today here in the United States.
The PKD Foundation would like to extend its gratitude to Otsuka for its steadfast support. "This was an unpaved road. Otsuka really cleared the path for more treatments on the horizon," said Alexis Denny, Director of Government Affairs for the PKD Foundation.
Betts added, "Many thanks to all of the patients who graciously took the time and resources to participate in the clinical trials to bring JYNARQUE™ to the PKD community. This treatment would not exist without these patients."
"We hope that this is just the beginning of new treatments on the horizon for patients suffering from PKD. We will continue to stand beside PKD patients until there is a cure, supporting them with access to future studies, to new treatments, and to ensure the affordability of care," said Betts.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use and medical devices. The agency is also responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
About the PKD Foundation
The PKD Foundation has been dedicated since its founding in 1982 to supporting and improving the lives of patients affected by polycystic kidney disease. These efforts are accomplished through promoting research to find treatments and a cure, as well as providing education, advocacy and awareness on a national level. The Foundation provides direct services to local communities nationwide and is the largest private funder of PKD research.
The PKD Foundation is the only organization in the United States solely dedicated to finding treatments and a cure for PKD. Our mission: We give hope. We fund research, advocate for patients and build a community for all affected by polycystic kidney disease (PKD). Our Vision: #endPKD
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SOURCE PKD Foundation