New Data from HELIOS-B Phase 3 Study Demonstrate Lower Rates of Gastrointestinal Events in ATTR-CM Patients Treated with Vutrisiran

Core Insights - Vutrisiran treatment resulted in a 37-49% reduction in gastrointestinal (GI) events in patients with ATTR-CM compared to placebo, demonstrating its efficacy and safety across various patient groups [1][2][4] Group 1: Efficacy of Vutrisiran - A 42% lower rate of GI events was observed in the overall population treated with vutrisiran compared to placebo [2] - In the vutrisiran monotherapy group, a 37% reduction in GI events was noted, while a 49% reduction was seen in patients who were on tafamidis at baseline [2] - Significant reductions of over 50% in individual GI symptoms such as diarrhea, nausea, and vomiting were reported across all study populations [2] Group 2: Safety Profile - The analysis indicated that the lower rate of GI events was consistent across hereditary and wild-type patients throughout the double-blind period [2] - Vutrisiran demonstrated a statistically significant 32% reduction in the risk of all-cause mortality and recurrent cardiovascular events compared to placebo in a censored monotherapy population [4] Group 3: Clinical Implications - The findings highlight the potential of vutrisiran as a first-line treatment for ATTR-CM, addressing the multisystem nature of the disease [4][5] - The results reinforce the strong monotherapy profile of vutrisiran, suggesting meaningful impacts on patient quality of life and overall health outcomes [5][6] Group 4: Regulatory and Market Context - Vutrisiran has received approvals in multiple regions, including the US, EU, and Japan, for treating both cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis [6][12] - The drug has accumulated over 8,000 patient-years of experience globally, establishing its position in the market [6]