New Long-Term Results for GIVLAARI® (givosiran) at the 62nd American Society of Hematology Annual Meeting & Exposition

05 Dec, 2020 New Long-Term Results for GIVLAARI® (givosiran) at the 62nd American Society of Hematology Annual Meeting & Exposition

During the 62^nd American Society of Hematology Annual Meeting & Exposition, we presented new 18-month interim results from the ongoing open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) in patients with acute hepatic porphyria (AHP), demonstrating sustained efficacy and safety with long-term dosing.

Kuter, et al. Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open-Label Extension

Eighteen-month interim results from the OLE period showed that long-term GIVLAARI treatment led to sustained lowering of ALA and PBG levels, and sustained reductions in the annualized rate of composite porphyria attacks and hemin use.

Long-term GIVLAARI treatment also led to improvements in multiple measures of QOL, and reductions in workdays missed due to AHP. The safety profile of GIVLAARI at 18 months remained acceptable and consistent with that previously observed.