Tolvaptan (Jinarc) Cleared in Europe for Rare Kidney Disease

Megan Brooks


May 27, 2015

The European Commission has approved tolvaptan (Jinarc, Otuska) for treatment of autosomal dominant polycystic kidney disease (ADPKD) in adults with stage 1 to 3 chronic kidney disease at the outset and evidence of rapidly progressing disease, the company announced today.

ADPKD is a rare chronic and progressive inherited disease affecting roughly 4 in 10,000 people in the European Union. ADPKD causes cyst proliferation and growth in the kidneys, leading to an increase in kidney size and complications that include chronic and acute pain, hypertension, and kidney failure.

Tolvaptan, a vasopressin-2 receptor antagonist, is the first drug therapy to be licensed in Europe for the treatment of the underlying pathophysiology of ADPKD.

The European Medicines Agency's Committee for Medicinal Products for Human Use recommended approval of tolvaptan for ADPKD in February 2015. At the time, the agency said there is a "clear unmet need for an effective therapy for ADPKD."

"Until now, healthcare professionals have focused on treating the signs and symptoms of ADPKD, with no specific treatment available to treat the disease," Ron T. Gansevoort, MD, PhD, University Medical Centre Groningen, the Netherlands, an expert in the field of polycystic kidney disease, said in an Otuska news release.

"Tolvaptan represents a significant medical breakthrough in the management of ADPKD. For the first time, healthcare professionals can modify the progression of the disease and preserve kidney function, with the potential to improve patients' quality of life and long-term outcomes," he added.

The approval is based on results of the phase 3 TEMPO 3:4 study involving 1445 adults with rapidly progressing early ADPKD from 129 sites worldwide.

Over the course of 3 years, the rate of total kidney volume increase was significantly less for tolvaptan-treated patients than for placebo-treated patients: 2.80% per year vs 5.51% per year, respectively. These data demonstrate an approximate 50% significant reduction in the annual increase in total kidney volume vs placebo, the company says.

Tolvaptan also showed a statistically significant reduction in the risk of multiple events of worsening kidney function, kidney pain, hypertension or albuminuria. Worsening kidney function was 61.4% less likely with tolvaptan than with placebo, the company notes, and medically significant kidney pain was 35.8% less likely in tolvaptan-treated patients.

As a result of a higher incidence of serious liver adverse effects with tolvaptan than placebo (2.3% vs 1.0%), patients taking tolvaptan will have to undergo monthly blood tests for the first 18 months of treatment with tolvaptan, and every 3 months thereafter, to mitigate this risk.

"Tolvaptan treatment must be initiated and monitored under the supervision of physicians with expertise in managing ADPKD and a full understanding of the risks of tolvaptan therapy including hepatic toxicity and monitoring requirements," the company notes.


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