Trials of genetic heart failure drug halted after patient deaths

John Maraganore, chief executive officer at Alnylam Pharmaceuticals

Alnylam Pharmaceuticals has discontinued development of an experimental therapy for a rare genetic condition that can cause heart failure because of higher death rates in patients taking the drug in late stage trials.

Revusiran, an investigational RNA interference (RNAi) therapeutic, was being developed to treat hereditary amyloidosis with cardiomyopathy, a condition in which amyloid plaque — similar to the substance found in the brains of Alzheimers’ patients — collects in organs including the heart, where it can cause heart failure.

Following recent reports of new onset or worsening peripheral neuropathy with revusiran in the phase II open-label study, the company asked the ENDEAVOUR Data Monitoring Committee (DMC) to review the data on an unblinded basis. The DMC did not find conclusive evidence for drug-related neuropathy, but informed the company that the benefit-risk profile for revusiran no longer supported continued dosing. A subsequent review of the unblinded ENDEAVOUR data by Alnylam Pharmaceuticals revealed higher rates of mortality in the revusiran arm than the placebo.

John Maraganore, chief executive officer at Alnylam, says: “Patient safety comes first. We have stopped all dosing and are actively monitoring patients across revusiran studies to ensure their safety. We will also continue to evaluate ENDEAVOUR data to understand the potential cause of these findings.”

Last updated
The Pharmaceutical Journal, PJ, October 2016, Vol 297, No 7894;297(7894):DOI:10.1211/PJ.2016.20201803

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