The US Food and Drug Administration (FDA) has approved United States-based Alnylam Pharmaceuticals' GIVLAARI (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP), a family of ultra-rare, genetic diseases characterised by debilitating, potentially life-threatening attacks, it was reported yesterday.
The product received approval in less than four months after acceptance of the NDA and was based on positive results from the ENVISION Phase 3 study, a randomised, double-blind, placebo-controlled, multinational study of 94 patients with acute hepatic porphyria, at 36 study sites in 18 countries – the largest ever interventional study conducted in AHP. In ENVISION, AHP patients on GIVLAARI experienced 70% (95% CI: 60%, 80%) fewer porphyria attacks compared to placebo. The product also resulted in a similar decrease in intravenous hemin use, and decrease in urinary aminolevulinic acid and urinary porphobilinogen.
The product is likely to be offered for shipment to healthcare providers in the United States by the end of 2019.
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