These are exciting times for the porphyria patient community: After the recent FDA approval of afamelanotide to treat patients with EPP, today, the US agency approved givosiran for the treatment of adult patients with acute hepatic porphyria (AHP). Prior to today’s approval, therapeutic options available to patients with AHP were only able to partially manage the recurrent acute and painful attacks that patients with AHP can suffer. With givosiran, patients now have a new option, which helps significantly reduce the number of attacks that require urgent medical interventions. The IPPN is delighted by today’s news and looks forward to givosiran being made broadly available to patients in need and to further approvals in more parts of the world.
