FDA Approves Attruby (Acoramidis) for ATTR-CM, Showing Near-Complete TTR Stabilization
核心洞察
The FDA has approved Attruby (acoramidis) for adults with transthyretin (搜索)-mediated amyloid cardiomyopathy (ATTR-CM (搜索)) to reduce cardiovascular death and hospitalization.
Attruby is the first and only approved product with a label specifying near-complete stabilization of transthyretin (搜索) (TTR (搜索)), addressing the root cause of ATTR-CM (搜索).
Clinical trials demonstrated Attruby's rapid benefit, with a 42% reduction in all-cause mortality and cardiovascular-related hospitalizations at 30 months compared to placebo.
BridgeBio Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Attruby™ (acoramidis) for the treatment of adult patients with transthyretin (搜索) amyloid cardiomyopathy (ATTR-CM (搜索)) to reduce cardiovascular death and cardiovascular-related hospitalization. The approval is based on the positive results from the ATTRibute-CM Phase 3 study.
Clinical Efficacy and Safety
The ATTRibute-CM Phase 3 study enrolled 632 participants with symptomatic ATTR-CM (搜索). Participants were randomized 2:1 to receive Attruby or placebo for 30 months. The trial met its primary endpoint, demonstrating a statistically significant reduction in all-cause mortality (ACM) and cardiovascular-related hospitalization (CVH) with a Win Ratio of 1.8 (p<0.0001). Attruby also demonstrated a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and 6-minute walk test. The increase in NT-proBNP on treatment was about half that of placebo.
According to Martha Grogan, M.D., of the Mayo Clinic, Attruby reduces all-cause mortality and cardiovascular hospitalization as early as three months after initiation of therapy.
In terms of safety, diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were more frequently reported in patients treated with Attruby versus placebo. These adverse reactions were generally mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between the Attruby and placebo groups (9.3% and 8.5%, respectively).
Mechanism of Action
Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR (搜索). Attruby was designed to mimic a naturally occurring "rescue mutation" of the TTR gene (T119M) that targets the root cause of ATTR-CM (搜索), destabilization of the native TTR tetramer. Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.
Patient Support and Access
BridgeBio offers a patient support services program, ForgingBridges™, for people in the U.S. prescribed Attruby and their families to receive help accessing this new therapy. More information about BridgeBio’s patient support services program is available on ForgingBridges.com or by calling 1-888-55-BRIDGE (1-888-552-7434).
Future Plans
BridgeBio submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM (搜索) in Europe.