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Otsuka finally gets FDA OK for kidney disease drug

Approval at last for rare disease treatment

OtsukaAt the second attempt, Otsuka has been given a green light in the US for tolvaptan, the first drug to be FDA-approved for polycystic kidney disease.

The FDA turned down Otsuka’s earlier marketing application for tolvaptan in 2013 due to a lack of clinical evidence that it worked, to the consternation of US patients with the rare and life-threatening genetic disease. Now – thanks to an additional clinical study – the Japanese drugmaker has the go-ahead to market the drug for adult patients with rapidly progressing, autosomal dominant PKD.

Otsuka – which will sell tolvaptan under the Jynarque brand name in the US – says the drug is the first treatment to slow the decline in kidney function in these patients, who have the most common form of PKD. It is already available for PKD in a number of markets outside the US, including Europe where it was approved in 2015 and is sold as Jinarc.

The Japanese drugmaker also sells another formulation of the drug under the Samsca brand for the treatment of low blood levels of sodium (hyponatraemia), and the tolvaptan range as a whole is one of its fastest-growing products, with sales rising around a third last year to reach 64.2bn yen (around $590m) last year. Otsuka said recently it expects US approval for PKD to drive sales up to 81bn yen this year.

The approval has been trumpeted by the PKD Foundation, which it says is a “historic day” for US patients, whose treatment options until now were limited to dialysis or a kidney transplant.

“For the past 35 years, our goal has been to support PKD patients from care to cure,” said the charity’s chief executive Andy Betts. “It is gratifying to play a part in the discovery of this treatment and to see it come to fruition.”

PKD disease is characterised by uncontrolled growth of cysts in the kidneys and other organs and can lead to kidney failure, and affects around 140,000 people in the US and millions worldwide. Tolvaptan is a vasopressin V2-receptor antagonist designed to inhibit the formation, proliferation and growth of those cysts.

In the additional REPRISE study, the drug was shown to reduce the decline in kidney function using the glomerular filtration rate (GFR) in the kidney as a biomarker by around 35% compared to placebo over the course of a year.

Data from the earlier TEMPO 3:4 study showed that Otsuka’s drug was able to reduce the total kidney volume in autosomal dominant PKD patents, as well as the time to progression of kidney disease (measured using creatinine levels) and symptoms such as kidney pain and hypertension.

Article by
Phil Taylor

25th April 2018

From: Regulatory



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