Alnylam announces approval of Givlaari (givosiran) in the European Union for the treatment of acute hepatic porphyria in adults and adolescents

Alnylam Pharmaceuticals

3 March 2020 - Givlaari approved based on ENVISION Phase 3 study results showing a 74% reduction in the rate of porphyria attacks.

Alnylam Pharmaceuticals today announced that the European Commission has granted marketing authorization for Gilvaari (givosiran), an injection for subcutaneous use targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older. 

AHP is an ultra-rare condition in which patients can experience debilitating attacks of severe abdominal pain, vomiting and seizures, which can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks. Many patients also experience chronic symptoms, including pain, which continue to be present between attacks.

Read Alnylam Pharmaceuticals press release

Michael Wonder

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Michael Wonder

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Outcome , Europe , Gene therapy