10 Jun, 2020 ENVISION Phase 3 Results for Givosiran Published in the “New England Journal of Medicine”
The New England Journal of Medicine (NEJM) published pivotal results from the ENVISION Phase 3 study of givosiran, our RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria, in a manuscript titled “Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.”
The data reported in the ENVISION Phase 3 study publication demonstrated that treatment with givosiran resulted in a significant and clinically meaningful reduction of 74% in the primary endpoint of the annualized rate of composite porphyria attacks (AAR) compared to placebo. Reductions were also observed in a number of secondary endpoints, including urinary ALA and PBG levels, days of IV hemin use and patient-reported daily worst pain. Patients treated with givosiran also reported favorable effects on exploratory endpoints related to use of analgesics, overall health status and activities of daily living. Key safety findings included elevations of serum aminotransferases, changes in serum creatinine/estimated glomerular filtration rate, and injection-site reactions. Givosiran demonstrated an encouraging and monitorable safety profile in AHP, a serious orphan disease that can be life threatening.