Following the presentation of detailed Phase III data last week, Alnylam Pharmaceuticals Inc.’s Amvuttra seems likely to become the first gene silencer approved for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). But perhaps not the last: Ionis Pharmaceuticals, Inc. and AstraZeneca PLC are awaiting pivotal data on Wainua (eplontersen) in the disease, and their decision to enroll huge numbers of patients could give their product an edge.
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