People living with ADPKD in England and Wales will now have access to JINARC® (tolvaptan), the first-ever treatment shown to slow progression of the life-threatening condition. NICE today released final guidance recommending tolvaptan in ADPKD for adults with chronic kidney disease (CKD) stages 2 or 3 at the start of treatment with evidence of rapidly progressing disease. Tolvaptan is the first disease-modifying ADPKD treatment available on the UK National Health Service (NHS).
ADPKD is the most common inherited kidney disease and is thought to affect 60,000 people in the UK and 205,000 people in Europe.The disease is primarily characterised by the development and enlargement of multiple fluid-filled cysts in the kidneys which lead to an increase in kidney size and complications that include chronic and acute pain, hypertension and kidney failure. Approximately half 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.”
Tolvaptan data evaluated by NICE included the results of the pivotal 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 approximately half (49%, p <0.0001) in the annual increase in total kidney volume for tolvaptan-treated patients versus those taking placebo. Furthermore, 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).10 Side effects associated with the way the drug works, e.g. thirst, polyuria, pollakiuria were observed in patients taking tolvaptan. 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 (March 2014) and Canada (February 2015). The NICE final guidance on tolvaptan follows its European marketing authorisation, granted in May 2015. Otsuka will continue to work with other European healthcare bodies to ensure that eligible patients have timely access to tolvaptan.
