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Alnylam Reports Positive Result From Interim Analysis Of Phase 3 Givosiran Study


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(RTTNews.com) - Alnylam Pharmaceuticals, Inc. ( ALNY ) announced positive topline results from the interim analysis of the ENVISION Phase 3 Study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria. The pre-specified interim analysis was based on lowering of urinary ALA levels as a surrogate biomarker that is reasonably likely to predict clinical benefit. The company said the results of the interim analysis showed that givosiran treatment was associated with a statistically significant reduction in urinary ALA levels in acute intermittent porphyria patients, relative to placebo.

The company plans to discuss the data and the regulatory path forward with the FDA, and, pending the outcome of those discussions, intends to file a New Drug Application (NDA) at or around year-end 2018 in support of a potential Accelerated Approval.


Read the original article on RTTNews (http://www.rttnews.com/2938684/alnylam-reports-positive-result-from-interim-analysis-of-phase-3-givosiran-study.aspx)


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This article appears in: World Markets , Stocks , Technology
Referenced Symbols: ALNY



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