Givosiran: A Review in Acute Hepatic Porphyria

Givosiran (Givlaari(R)) is an delta-aminolevulinic acid synthase 1 (ALAS1)-directed small interfering RNA (siRNA) approved for the treatment of acute hepatic porphyria (AHP). In the phase 3 ENVISION trial, givosiran significantly reduced the annualized rate of composite porphyria attacks (i.e. attacks requiring hospitalization, urgent healthcare visit or intravenous hemin administration at home) compared with placebo in patients with recurrent acute intermittent porphyria (the most common type of AHP) attacks. Givosiran also improved several other outcomes, including hemin use and pain (the cardinal symptom of AHP). While generally well tolerated with an acceptable safety profile, the drug may increase the risk of hepatic and kidney adverse events. Givosiran offers the convenience of once-monthly subcutaneous administration. Available evidence indicates that givosiran is an important newer therapeutic option for patients with AHP and severe recurrent attacks.

Automated summary

Givosiran is an approved treatment for AHP, significantly reducing attack rates and improving symptoms in patients. Despite a risk of adverse events, it offers the convenience of subcutaneous administration and is an important new therapeutic option for AHP patients with severe recurrent attacks.