Treatment for inherited kidney disease approved by NICE

Tolvaptan (Jinarc) has been accepted by NICE for treating autosomal dominant polycystic kidney disease (ADPKD) in adults.

ADPKD is the most common inherited kidney condition and is characterised by small, fluid-filled cysts in the kidneys. | STEVE GSCHMEISSNER/SCIENCE PHOTO LIBRARY
ADPKD is the most common inherited kidney condition and is characterised by small, fluid-filled cysts in the kidneys. | STEVE GSCHMEISSNER/SCIENCE PHOTO LIBRARY

Jinarc is indicated in the treatment of ADPKD in patients with chronic kidney disease stage 1 to 3 and evidence of rapidly progressing disease.

NICE recommends the use of tolvaptan as an option to slow the progression of cyst development and renal insufficiency only if:

  • the patient has chronic kidney disease stage 2 or 3 at the start of treatment
  • there is evidence of rapidly progressing disease, and
  • the company provides the drug with the discount agreed in the patient access scheme

Further information

Tolvaptan is a selective vasopressin V2 receptor antagonist. Cell proliferation, cyst formation and fluid excretion are reduced with tolvaptan treatment, slowing kidney growth and protecting kidney function. Tolvaptan is the only approved disease-modifying therapy for ADPKD.

There is a risk of liver injury in patients treated with tolvaptan and prescribers must test hepatic transaminase and bilirubin levels before starting treatment, monthly for the first 18 months of treatment, and at 3-month intervals thereafter. Patients should also be monitored for symptoms that may indicate liver injury.

The most common adverse effects of tolvaptan are thirst, headache, dizziness, GI upset, dry mouth, increased urination and fatigue.

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