Alnylam says givosiran meets primary, majority of secondary endpoints in study

Alnylam Pharmaceuticals announced that the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 in development for the treatment of acute hepatic porphyria, met its primary efficacy endpoint and the majority of secondary endpoints. Specifically, givosiran met the primary endpoint of reduction in the annualized rate of composite porphyria attacks relative to placebo and achieved statistically significant results for five of nine secondary endpoints, with a safety and tolerability profile that the company believes is encouraging, especially in this high unmet disease. Based on these results, the company plans to complete its rolling submission of a New Drug Application and file a Marketing Authorization Application in mid-2019. Givosiran has received Breakthrough Therapy and Prime designation by the FDA and EMA, respectively, and has Orphan Drug status in the U.S. and EU for the treatment of AHP. The Phase 1 results of givosiran were recently published in The New England Journal of Medicine. Full ENVISION study results will be presented in an oral plenary session on Saturday, April 13 at EASL in Vienna, Austria. The ENVISION results have not yet been reviewed by regulatory authorities.
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