21 October 2021 - NICE have recommended the use of givosiran, a gene silencing therapy, on the NHS in England, as an option for the treatment of acute hepatic porphyria, a painful disease usually first seen in young women in their 20s.

Givlaari (givosiran), uses ‘gene silencing’ RNA interference technology, to target the production of pathogenic compounds in people with acute hepatic porphyria. NICE’s approval is based on data from a Phase 3 trial, in which treatment led to a 74% reduction in the rate of porphyria attacks compared to placebo.

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