For the first time, a treatment will be available on the NHS in England and Wales that can delay the development of autosomal dominant polycystic kidney disease (ADPKD).
The National Institute for Health and Care Excellence has recommended the drug tolvaptan (Jinarc) be made available for treating ADPKD, which is thought to affect around 60,000 people in the UK.
The approval covers use of the drug in adults with stage 2 or 3 chronic kidney disease at the start of treatment and with evidence of rapidly progressing disease.
“Until now there hasn’t been a treatment that can delay the progression of ADPKD”
Tolvaptan, manufactured by Otsuka Pharmaceuticals, is a treatment option for slowing the progression of cyst development and helping protect renal function.
A selective vasopressin antagonist, tolvaptan inhibits the binding of vasopressin to the V2 receptors, said the NICE guidance, which was published today.
It reduces cell proliferation, cyst formation and fluid excretion, which in turn reduces kidney growth and protects kidney function.
Where NICE recommends a treatment, the NHS in England andf Wales is legally obliged to begin funding the drug within three months of the final publication date.
Tolvaptan is taken orally, twice daily as a split dose. Doses can be titrated according to tolerability up to a maximum total daily dose of 120 mg.
It is available as 15mg, 30mg, 60mg and 90mg tablets, in 28-day packs of split-dose tablets, at a flat net price of £1,208.20, equating to £43.15 per day, regardless of dose.
Tess Harris, chief executive of the PKD Charity said: “ADPKD is the most common, life-threatening genetic kidney disease with an unpredictable and often cruel impact on the families it affects.
“Until now there hasn’t been a treatment that can delay the progression of ADPKD,” said Ms Harris.
“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,” she added.