For the last 20 years Alnylam Pharmaceuticals has converted Nobel-winning research on RNA-based gene-silencing into medicines, winning approvals for drugs delivered to the liver. That’s where some misfolded proteins are made, and in an increasingly diagnosed heart disease called ATTR cardiomyopathy, they eventually jam up muscles in the heart and contribute to heart failure. But ATTR isn’t the company’s only target.
Alnylam’s experimental heart drug vutrisiran significantly slashed the risk of death and serious cardiovascular complications among patients with this heart disease, late-stage clinical trials showed. Applications seeking approval in the U.S. and Europe have been submitted, with decisions likely coming next year. The drug is already approved under the brand name Amvuttra in a similar disease called ATTR polyneuropathy, but ATTR-CM affects more people, with estimates reaching 300,000 patients around the world.
“The job at hand here is to make sure that patients can get diagnosed early and treated early,” Alnylam CEO Yvonne Greenstreet said Thursday at the STAT 2024 Summit in Boston. “Competition is a good thing because it actually brings more education to physicians.”
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