New Treatment for Patients with Acute Intermittent Porphyria

Acute intermittent porphyria is an inherited disease of heme group hepatic biosynthesis. A promising Phase I clinical trial for its treatment has recently been published in the prestigious New England Journal of Medicine. The new drug, givorisan, interferes with RNA and prevents the synthesis of neurotoxic intermediate metabolic products that result in recurrent attacks of abdominal pain. Monthly injections of givosiran significantly reduced the number of crises compared to placebo.
N Engl J Med 2019; 380:549-558