Givosiran to treat acute hepatic porphyria in US

February 6, 2020 0 By FM

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. 

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. Givosiran also received Orphan Drug designation, Alnylam Pharma said.