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ENVISION Phase III study results of Givlaari to treat acute hepatic porphyria were published online in the NEJM.- Alnylam Pharma
Alnylam Pharmaceuticals, Inc. announced that pivotal results from the ENVISION Phase III 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 FDA 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 III Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria,” will appear in the June 11, 2020 issue of NEJM.
The data reported in the ENVISION Phase III 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.
See-"Phase III Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria"- Manisha Balwani, M.D., Eliane Sardh, M.D., Ph.D., Paolo Ventura, M.D., Paula Aguilera Peiró, M.D., David C. Rees, F.R.C.P., Ulrich Stölzel, M.D.,., et al., for the ENVISION Investigators-June 11, 2020.N Engl J Med 2020; 382:2289-2301. DOI: 10.1056/NEJMoa1913147.
Condition: Porphyria
Type: drug