Alnylam Pharmaceuticals Inc. stands to significantly increase the addressable population for Amvuttra (vutrisiran) with positive Phase III data in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). However, without detailed data from the clinical trial, the commercial prospects remain unclear given the lengthened follow-up in the study and the increasingly competitive market.
The Cambridge, MA-based biotech announced topline results from the HELIOS-B study in ATTR-CM on 24 June, stating that the study met its primary endpoint, a composite of all-cause mortality and recurrent cardiovascular events during the double-blind period in the overall population – including those who received
Key Takeaways
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Alnylam plans to file a supplemental approval application with the FDA to expand the label for Amvuttra after positive results from the HELIOS-B trial in ATTR-CM.
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The company projects a tenfold increase in the addressable patient population for the drug, which brought in $195m in revenue in the first quarter