The US Food and Drug Administration (FDA) has approved Attruby (acoramidis), marking a significant advancement for patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This progressive disease occurs when misfolded transthyretin (TTR) proteins form amyloid deposits in the heart, leading to cardiac dysfunction. Attruby offers a near-complete stabilization of TTR — over 90 percent — halting the formation of amyloid fibrils that cause tissue damage and addressing the underlying protein instability that drives disease progression.
Attruby offers a more potent option, following Pfizer’s tafamidis (Vyndaqel and Vyndamax), the first therapy and TTR stabilizer approved to treat ATTR-CM.
ATTR-CM has been historically difficult to manage, with limited therapeutic options. Dr. Martha Grogan of the Mayo Clinic noted in BridgeBio’s news release that ATTR-CM is a life-threatening condition with a poor prognosis when untreated.
The FDA’s decision follows compelling data from the ATTRibute-CM Phase III study, which enrolled 632 symptomatic patients with either wild-type or hereditary ATTR-CM. Participants were randomized to receive acoramidis or placebo over 30 months.
The trial demonstrated a 42 percent reduction in combined cardiovascular death and recurrent hospitalizations at Month 30, with cardiovascular-related hospitalizations reduced by half. Clinical markers, including NT-proBNP and 6-minute walk distance, also improved substantially. Notably, acoramidis delivered measurable benefits as early as three months into treatment, making it the most rapid response observed in Phase III trials for ATTR-CM to date.
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Patient advocacy groups, including the Amyloidosis Support Groups led by Muriel Finkel, highlight the transformative impact of Attruby on the lives of those with ATTR-CM. The availability of a treatment that addresses both survival and quality of life represents a long-awaited milestone for patients and their caregivers.
From a regulatory standpoint, BridgeBio has taken steps to ensure global access to Attruby. A Marketing Authorization Application has been submitted to the European Medicines Agency (EMA), with a decision anticipated in 2025. BridgeBio has also partnered with Bayer for commercialization efforts in Europe.
Domestically, BridgeBio has launched the ForgingBridges program, offering patient support services such as insurance navigation, financial assistance and treatment access.
BridgeBio is also working to address gaps in ATTR-CM diagnosis through its TRACE-AI Network Study, a collaboration with the Cardiovascular Data Science Lab (CarDS Lab) at the Yale School of Medicine. This initiative employs advanced artificial intelligence tools to identify undiagnosed ATTR-CM cases across diverse health systems, focusing on improving early detection in underserved populations.
BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.
BridgeBio’s leadership, including Neil Kumar, emphasizes the significance of Attruby’s approval as a cornerstone in their mission to develop therapies for genetic diseases. Looking ahead, the company plans to pursue regulatory approvals in Japan and Brazil, further expanding access to this life-changing treatment.
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