Randomized trial reveals reduced porphyria attacks with givosiran in acute intermittent porphyria, indicating therapeutic efficacy alongside hepatic and renal risks.
Among patients with acute intermittent porphyria, those who received givosiran had a significantly lower rate of porphyria attacks and better results for multiple other disease manifestations than those who received placebo. The increased efficacy was accompanied by a higher frequency of hepatic and renal adverse events. (Funded by Alnylam Pharmaceuticals; ENVISION ClinicalTrials.gov number, NCT03338816.).
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Balwani et al. (2020) studied this question.
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