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Alnylam Announces Publication of ENVISION Phase 3 Study Results for Givosiran in the New England Journal of Medicine
− The ENVISION Phase 3 Study Evaluated the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyria (AHP) − − Givosiran Demonstrated
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n− The ENVISION Phase 3 Study Evaluated the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyria (AHP) −\n\n\n− Givosiran Demonstrated Significant Reduction in the Rate of Porphyria Attacks Compared with Placebo in Patients with AHP –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that pivotal results from the ENVISION Phase 3 study of givosiran, an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), were published online in the New England Journal of Medicine (NEJM). GIVLAARI® (givosiran) was approved by the U.S. Food and Drug Administration for the treatment of adults with AHP in November 2019, marking the first ever approval of a GalNAc-conjugate RNAi therapeutic—a landmark in the advancement of precision genetic medicines. It also received marketing authorization from the European Commission in March 2020 for the treatment of AHP in adults and adolescents aged 12 years and older. The full manuscript, titled “Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria,” will appear in the June 11, 2020 issue of NEJM.\n\n\nThe data reported in the ENVISION Phase 3 study publication demonstrated that RNAi-mediated targeting of liver ALAS1 by givosiran led to sustained reductions in aminolevulinic acid (ALA) and porphobilinogen (PBG)—the toxic heme synthesis intermediates believed to be causal for the disease manifestations of AHP. Relative to placebo, treatment with givosiran resulted in a significant and clinically meaningful reduction of 74 percent in the primary endpoint of the annualized rate of composite porphyria attacks (AAR), defined as those attacks requiring hospitalization, urgent healthcare visit, or intravenous hemin administration at home, in patients with acute intermittent porphyria (AIP), the most common form of AHP, over six months.\n\n\nImprovements were also observed in a number of secondary endpoints (assessed in patients with AIP), including reductions in urinary ALA and PBG levels, days of intravenous hemin use and daily worst pain. Additionally, in all patients with AHP, mean AAR was significantly reduced by 73 percent relative to placebo. Patients treated with givosiran also reported favorable effects on ex...