CHMP Backs Tolvaptan (Jinarc) for Rare Kidney Disease

Megan Brooks

Disclosures

February 27, 2015

The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of tolvaptan (Jinarc, Otuska) to slow progression of cyst development and failing kidney function in adults with autosomal dominant polycystic kidney disease (ADPKD).

Tolvaptan is indicated for patients with ADPKD with chronic kidney disease stage 1 to 3 at initiation of treatment with evidence of rapidly progressing disease.

ADPKD is a chronic and progressive inherited disease affecting roughly 4 in 10,000 people in the European Union (EU). "There is therefore a clear unmet need for an effective therapy for ADPKD," the EMA says in a statement.

Tolvaptan, a vasopressin-2-receptor antagonist, would be the first drug specifically approved in the EU for ADPKD.

"In ADPKD, it is thought that kidney cells do not respond normally to vasopressin, leading to the formation of fluid-filled cysts. By blocking vasopressin receptors in the kidneys, Jinarc can slow down cyst formation," the EMA says.

The positive opinion is based on a 3-year phase 3 study involving 1445 adults with ADPKD. Compared with placebo, tolvaptan demonstrated a statistically significant 49% reduction in the annual increase in total kidney volume and a 30% reduction in the decline in kidney function.

Because of a higher incidence of serious liver adverse effects with tolvaptan than placebo (2.3% vs 1.0%), the CHMP recommended additional monitoring for tolvaptan-related liver damage. "Although no cases of liver failure were found in this study, it is possible that in a wider population of patients with ADPKD tolvaptan may cause liver injury that could progress to liver failure," the EMA notes.

The CHMP would like tolvaptan to be prescribed in the context of a registry to allow for additional monitoring, including blood tests to check the patient's liver function before starting treatment and then repeated every month for 18 months and every 3 months thereafter. A postapproval safety study will provide additional safety data.

Tolvaptan "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 liver damage, and monitoring requirements," the EMA says.

The CHMP opinion will now be sent to the European Commission for the adoption of a decision on EU-wide marketing authorization.

Tolvaptan is already approved in the EU for treating hyponatremia.

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