Kidney diseaseNICE has issued final guidance supporting the use of tolvaptan (Jinarc) for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

It means tolvaptan is the first approved pharmaceutical therapy available in Europe for the treatment of the disease, which is the most common, life-threatening genetic kidney disease in the UK.

The recommendation means that adults with stage 2 or stage 3 ADPKD will be able to receive treatment for the disease, which causes cyst proliferation and growth in the kidneys, leading to an increase in kidney size and resulting in complications that include chronic and acute pain, hypertension and kidney failure, necessitating dialysis or kidney transplant. About 50% of people with ADPKD will require a kidney transplant or dialysis by the age of 54.

“ADPKD is the most common, life-threatening genetic kidney disease with an unpredictable and often cruel impact on the families it affects,” said Tess Harris, Chief Executive of the PKD Charity. “Until now there hasn’t been a treatment that can delay the progression of ADPKD. Today’s NICE guidance has the potential to fundamentally change the outlook for patients with ADPKD and their families and brings great hope for the future.”

NICE approval follows the results of the Phase III TEMPO 3:4 Trial – the largest study ever undertaken in ADPKD. Over a three-year period, the study demonstrated a significant reduction of about half (49%, p <0.0001) in the annual increase in total kidney volume for tolvaptan-treated patients versus those taking placebo. Tolvaptan was shown to reduce the rate of decline in kidney function compared to that of placebo-treated patients by approximately 32% (p <0.001).

Patients taking tolvaptan were also 36% less likely to experience clinically significant kidney pain compared to those taking placebo (p=0.007). Side effects associated with the way the drug works, e.g. thirst, polyuria and pollakiuria were observed in patients taking tolvaptan, and a risk of liver injury was also identified.

Dr Richard Sandford, Consultant Clinical Geneticist at Addenbrookes Hospital in Cambridge, commented: “For many people with ADPKD, kidney failure at a relatively young age has been a daunting prospect which we’ve been unable to guard against. They have often seen other close family members require dialysis or a kidney transplant. This NICE recommendation will give us the opportunity to help some patients with ADPKD to reduce the number of symptoms they experience and potentially slow their progression towards kidney failure.”

Tolvaptan was approved for people with ADPKD in Japan in March 2014 and Canada in February this year. The NICE final guidance on tolvaptan follows its European marketing authorisation, granted in May. Pharmaceutical Otsuka will continue to work with other European healthcare bodies to ensure that eligible patients have timely access to tolvaptan.