FDA Approves Otsuka’s Drug for Rare Kidney Disease

Rare Daily Staff

The U.S. Food and Drug Administration approved Otsuka Pharmaceutical Jynarque, the first drug treatment to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease or ADPKD, a rare genetic disease that can lead to dialysis or kidney transplantation.

“This approval is important news for many adults at risk of rapidly progressing ADPKD in the U.S., who have had no therapeutic alternatives to delay the eventual end-stage interventions of dialysis or kidney transplantation,” said Tatsuo Higuchi, president and representative director of Otsuka Pharmaceutical.

ADPKD 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 United States and impacts families across multiple generations, since a parent with ADPKD has a 50 percent chance of passing the disease on to each of their children.

Jynarque is a selective vasopressin V2-receptor antagonist indicated to slow kidney function decline in adults at risk of rapidly progressing ADPKD. The medication has been approved as a treatment for adults with ADPKD in Japan, the European Union, Canada, South Korea, Switzerland, Hong Kong, Australia, Turkey, and Taiwan.

The FDA’s approval of Jynarque is supported by data from two pivotal trials.

Jynarque can cause serious and potentially fatal liver injury, and acute liver failure requiring liver transplantation has been reported. It has been associated with elevations of blood alanine and aspartate aminotransferases, with infrequent cases of concomitant elevations in bilirubin-total. To ensure the safety of patients taking Jynarque, it is necessary to measure ALT, AST and bilirubin before initiating treatment, and monitoring these levels regularly.

The company said Jynarque will be sold in a 28-day treatment pack at a wholesale cost of $13,041.10.

“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.” said Michal Mrug, associate professor at the University of Alabama at Birmingham and investigator on the one of the pivotal trials. “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.”

April 25, 2018
Photo: Tatsuo Higuchi, president and representative director of Otsuka Pharmaceutical.