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FDA approves Jynarque for rapidly progressing autosomal dominant PKD
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The FDA has approved Jynarque as the first treatment in the U.S. for adult patients with rapidly progressing autosomal dominant polycystic kidney disease, the most common form of polycystic kidney disease.
Jynarque (Otsuka Pharmaceuticals) is also known as tolvaptan.
"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," Andy Betts, CEO of the PKD Foundation, said in a related press release. "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."
Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disease that can lead to dialysis or kidney transplantation. It is a progressively debilitating and often painful disorder in which fluid-filled cysts develop in the kidneys over time. These cysts enlarge the kidneys and impair their ability to function normally, leading to kidney failure in most patients. ADPKD is diagnosed in approximately 140,000 people in the U.S., and impacts families across multiple generations, since a parent with ADPKD has a 50% chance of passing the disease on to each of their children.
"The progressive nature of ADPKD means that kidney function gets worse over time, eventually leading to end-stage renal disease. This progression happens more rapidly for some patients than others," Michal Mrug, MD, associate professor at the University of Alabama at Birmingham and a study investigator of Jynarque, said in a related press release. "Today's approval is great news for adults at risk of rapidly progressing ADPKD because by slowing the decline in kidney function, this therapy may give them more time before kidney transplant or dialysis."
Jynarque will be available in the U.S. for patients in the coming weeks.
References: https://www.otsuka.co.jp/en/company/newsreleases/2018/20180425_1.html
Disclosures:
Healio Nephrology was unable to determine relevant financial disclosures at the time of publication.