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Jinarc backed for European approval in rare kidney disease

Positive opinion for Otsuka comes just before rare disease awareness day

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The European Medicines Agency has recommended granting a marketing authorisation to Otsuka's orphan drug Jinarc (tolvaptan). 

Jinarc is designed to slow the progression of cyst development and failing kidney function in patients with autosomal dominant polycystic kidney disease (ADPKD). 

The drug is specifically for use in patients with normal to moderately-reduced kidney function and that have rapidly progressing ADPKD.

ADPKD affects around 4 in 10,000 people in the European Union (EU) and is therefore classified as a rare disease - Jinarc has also been given orphan drug status to recognise its place as a treatment for an unmet medical need.

The decision to recommend the drug for a European licence comes auspiciously before tomorrow's Rare Disease Day, an annual event that looks to raise awareness about patients with rare diseases, and the need for new treatments.

The drug, a vasopressin-2-receptor antagonist, is already authorised in the EU for treating hyponatraemia (abnormally low sodium levels), although the doses studied in ADPKD are different.

For its new licence the CHMP, which assesses the safety and efficacy of new medicines in Europe, has recommended additional monitoring of the risk of liver damage with Jinarc, as recent studies found a greater number of people with serious liver adverse effects when taking Jinarc compared with placebo.

The CHMP says that while no cases of liver failure were found in Otsuka's latest trial, it “is possible that in a wider population of patients with ADPKD tolvaptan may cause liver injury that could progress to liver failure”.

Therefore it says that prescribing of the drug must be used alongside blood tests to assess the liver functions of patients during treatment. 

The European Commission will now make a final decision on the treatment, with this likely to come within the next three months. 

ADPKD is an inherited condition marked by the growth of numerous fluid-filled cysts in the kidneys and other organs. The growth of cysts eventually affects kidney function and can cause the kidneys to fail. Symptoms include abdominal pain, problems with urinating, high blood pressure and infection.

No medicine is specifically authorised in the EU to treat patients with ADPKD and current treatment focuses on the treatment of symptoms and complications. 

Article by
Ben Adams

27th February 2015

From: Sales



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