Alnylam Pharmaceuticals has secured U.S. Food and Drug Administration (FDA) approval for vutrisiran, a subcutaneous injection marketed as Amvuttra, to treat adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM). Vutrisiran was previously approved for treating polyneuropathy of hereditary ATTR amyloidosis (hATTR-PN).
The approval was supported by data from the HELIOS-B clinical trial, which demonstrated that vutrisiran significantly reduced all-cause mortality and recurrent cardiovascular events, while also improving quality of life for patients. The trial involved 655 participants, with treatment groups showing notable benefits compared to a placebo.
Yvonne Greenstreet, CEO of Alnylam, expressed gratitude to all involved in the trials, emphasizing that this approval marks a key milestone in their ongoing commitment to patients with ATTR amyloidosis. “We are not stopping here,” Greenstreet stated, indicating continued innovation for those affected by this condition.
Vutrisiran requires four subcutaneous doses per year, and while its annual cost is around $477,000, Alnylam has ensured that most patients will have insurance coverage similar to that provided for hATTR-PN.
With the addition of vutrisiran, the ATTR-CM treatment landscape has expanded, joining Pfizer’s tafamidis (Vyndaqel) and BridgeBio’s acoramidis (Attruby), providing more options for patients living with this challenging disease.
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