Otsuka Pharmaceutical’s drug Jynarque (tolvaptan) received US Food and Drug Administration (FDA) approval as the first drug treatment in the United States to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease (ADPKD). The drug is a selective vasopressin V2-receptor antagonist.
Tolvaptan showed a greater reduction in estimated glomerular filtration rate compared to placebo, meeting the primary endpoint of the REPRISE trial (Replicating Evidence of Preserved Renal Function: an Investigation of Tolvaptan Safety and Efficacy in ADPKD).
The data appeared in a late-breaking abstract at ASN Kidney Week 2017 and were simultaneously published online in the New England Journal of Medicine (1).
Tolvaptan caused “more elevations in aminotransferase and bilirubin levels,” according to the NEJM. “The efficacy and safety of tolvaptan in patients with later-stage ADPKD are unknown.” After tolvaptan therapy ended, the results were reversible for aminotransferase, and bilirubin levels did not exceed twice the upper limit of the normal bilirubin range, the NEJM noted.
“We are thrilled to be a part of this first milestone in treatment for ADPKD,” said PKD Foundation CEO Andy Betts. “For the past 35 years, our goal has been to walk with PKD patients every step of the way. It is gratifying to play a part in the inception of the discovery of this treatment, and to see it come to fruition. We hope that this is just the beginning of a new chapter for adults at risk of rapidly progressing ADPKD who suffer from the disease.”
Jynarque is available only through a restricted distribution program, according to FirstWord Pharma. Patients must have testing for blood alanine and aspartate aminotransferases levels, as well as bilirubin, before initiating treatment with tolvaptan. Patients also must be tested at two weeks and four weeks after treatment begins, as well as monthly for 18 months and every three months afterward.
The company noted that the drug will be sold in a 28-day treatment pack at a wholesale cost of $13,041.10.
Reference
Torres V , et al.. Tolvaptan in later-stage autosomal dominant polycystic kidney disease. N Engl J Med 2017; 377:1930–1942.
