Givosiran to treat acute hepatic porphyriaJanuary 6, 2020
The US FDA has granted approval to givosiran (Givlaari) for the treatment of adult patients with acute hepatic porphyria.
The approval of givosiran was based on the results of a clinical trial of 94 patients with acute hepatic porphyria. Patients received a placebo or givosiran.
Givosiran’s performance was measured by the rate of porphyria attacks that required hospitalisations, urgent health care visits or intravenous infusion of hemin at home.
Patients who received givosiran experienced 70% fewer porphyria attacks compared to patients receiving a placebo.
The FDA granted this application Breakthrough Therapy designation and Priority Review designation. Givlaari also received Orphan Drug designation.