Safety Concerns From Ionis' Pivotal Amyloidosis Trial Bolsters Alnylam's Program Outlook
Safety concerns from Ionis’ pivotal NEURO-TTR study evaluating inotersen (formerly IONIS-TTRRx) in familial amyloid polyneuropathy--a rare, hereditary disease that causes toxic buildup in the nervous system--sent Alnylam’s and Ionis’ stocks ricocheting in opposite directions. While Alnylam’s amyloidosis drug patisiran, which is also in pivotal testing and is expected to release top-line results this September, has its own side effect issues, we believe the string of severe adverse events with Io...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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