Alnylam is in the early stages of building a moat around its RNA-based drug platform.
While Alnylam’s fourth-quarter results were largely expected, updates on the company’s clinical trial timelines paint an optimistic picture for the next few years. The company’s lead candidate patisiran (familial amyloidotic polyneuropathy) could reach the market as early as 2018 if pivotal trial results and regulatory approval fall in Alnylam’s favor, with fitusiran (hemophilia) and givosiran (hepatic porphyrias)  following in subsequent years. However, we do not plan on changing our $50 per sh...Alnylam Pharmaceuticals is a commercial-stage biopharmaceutical company developing therapeutics based on RNA interference (RNAi). RNAi is a biological pathway within cells for sequence-specific silencing and regulation of gene expression. The company has two RNAi-based medicines, ONPATTRO? (patisiran) and GIVLAARI? (givosiran). ONPATTRO is approved by the United States Food and Drug Administration (FDA) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults and has also been approved in the European Union, Japan, Canada and Switzerland. GIVLAARI is approved by FDA for the treatment of adults with acute hepatic porphyria.
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