New Drug for Kidney Disease Approved for NHS Use

The National Institute for Health and Care Excellence (NICE) has approved a breakthrough drug for a potentially fatal type of kidney disease for NHS use. Trials have shown the drug to be capable of slowing down the disease’s progression.

Tolvaptan is the first ever treatment for autosomal dominant polycystic kidney disease (ADPKD) – an incurable disease that causes cysts to develop in the kidneys, and can cause heart attacks, stroke and kidney failure. The disease, which accounts for one in ten people on dialysis in the UK, can be very painful and debilitating, and sometimes requires surgical interventions.

Tolvaptan will be available from early 2016 as a twice-daily tablet which helps to slow the rate at which the cysts develop. A three-year trial also found it to reduce the rate of worsening kidney function, pain and infections.

The drug aims to make the disease more manageable and reduce the need for dialysis or transplant. NICE recommends it as a possible treatment for people with rapidly progressing ADPKD who have chronic stage 2 or stage 3 kidney disease at the start of their treatment.

“This drug will give us the opportunity to help some patients to reduce the number of symptoms they experience and potentially slow their progression towards kidney failure,” says Dr Richard Sandford, a consultant clinical geneticist at Addenbrooke’s Hospital, Cambridge.

Tess Harris, CEO of the Polycystic Kidney Disease Charity, has ADPKD and says: “Until now there hasn’t been a treatment that can delay its progression. This has the potential to fundamentally change the outlook for patients with ADPKD and their families, and brings great hope for the future. Tolvaptan is the first therapy to target the underlying disease and not just the management of symptoms and complications.”