First treatment for acute hepatic porphyria

11 février 2020 – EMA’s human medicines committee (CHMP) recommended to grant an EU marketing authorization for Givlaari (givosiran). Givlaari is the first treatment for acute hepatic porphyria (AHP) in adults and adolescents. AHP is a rare genetic condition in which patients lack certain enzymes needed to produce a basic structure of haemoglobin that binds to oxygen. It is characterized by an accumulation of porphyrins in the body to toxic amounts which causes severe abdominal pain, vomiting and nervous system disorders and may be fatal. For further information, see here.