UK – NICE recommends gene silencing therapy for porphyria patients on NHS

NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as an option for the treatment of acute haptic porphyria (AHP), a painful disease usually first seen in young women in their 20s.

AHP is a group of rare genetic diseases, characterised by painful and potentially life-threatening attacks. It is caused by a genetic defect, and people suffering from it lack the enzymes needed to produce haem, a basic structure of the blood protein haemoglobin.

Patients suffering from AHP may experience recurrent attacks which may result in hospitalisation. There are currently approximately 17 patients across the UK who are diagnosed each year…