FDA Approves Vutrisiran For Adult Patients With ATTR-CM
The decision makes the RNAi therapeutic the only therapy approved by the FDA to treat ATTR-CM and hATTR-PN.
The FDA has approved vutrisiran (Amvuttra) for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adult patients to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits, Alnylam Pharmaceuticals announced in a release.1 The decision makes vutrisiran the only therapy approved to treat both ATTR-CM and polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN).
FDA Approves Vutrisiran For Adult Patients With ATTR-CM / JHVEPhoto - stock.adobe.com
The approval of vutrisiran was based on data from the phase 3 HELIOS-B (NCT04153149) study, in which the therapy met its primary endpoint of composite of all-cause mortality and recurrent cardiovascular events up to month 36. The therapy also met all of its key secondary endpoints, including change from baseline in a 6-minute walk test and all-cause mortality. The data, which was presented at the European Society of Cardiology Congress, was also published in the New England Journal of Medicine.2
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Vutrisiran is an RNA interference (RNAi) therapy that’s delivered through 4 yearly subcutaneous doses. It significantly decreases deposition of misfolded transthyretin (TTR) fibrils by reducing their production, which can cause cardiovascular damage and premature death in patients with ATTR-CM.
“Despite recent advances, there remains a significant need for patients living with ATTR-CM and I’ve witnessed, firsthand, the impact that ATTR amyloidosis can have on families, including diminished quality of life and the loss of loved ones,” Muriel Finkel, president of the Amyloidosis Support Groups, said in a release.1 “The availability of this groundbreaking treatment option is a significant moment for patients living with ATTR amyloidosis. It represents a beacon of hope for our community.”
HELIOS-B is a randomized, double-blind, placebo-controlled, multicenter phase 3 clinical trial evaluating the efficacy and safety of vutrisiran compared to placebo in patients with ATTR-CM. The study cohort included 655 adult patients aged 18 to 85 years who had a diagnosis of ATTR-CM, evidence of cardiac involvement, and a clinical history of heart failure. Patients randomly received either vutrisiran 25 mg or placebo every 12 weeks for up to 36 months.
The study found vutrisiran reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% in the overall population. In the double-blind period of the study, the therapy reduced mortality by 31% and by 36% up to 42 months. Compared to placebo, treatment with vutrisiran resulted in a smaller decline in distance covered on the 6-minute walk test and in the KCCQ-OS score. Similar positive benefits of the therapy were also seen in the monotherapy group, with the risk of all-cause mortality and recurrent cardiovascular events reduced by 33%.
Additionally, the safety and tolerability of vutrisiran was consistent with previous data. The rate of adverse events was similar between the therapy and placebo groups. Serious adverse events occurred in 62% of the patients in the vutrisiran group and in 67% in the placebo group. Adverse events leading to discontinuation occurred in 10 patients in the vutrisiran group and 13 patients in the placebo group.
“This FDA approval provides an opportunity to further transform ATTR-CM treatment with a new mechanism of action,” Ronald Witteles, MD, an investigator on the HELIOS-B trial, said in a release.1 “The HELIOS-B clinical trial found that vutrisiran allowed patients to live longer, experience fewer hospitalizations, and improve how they function and feel. The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by vutrisiran’s ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression.”
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