1 ATTR-CM drug may best another for preserving heart health, analysis finds
Absent head-to-head trials, researchers use US insurance data to compare
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Attruby (acoramidis) may be better than Vyndamax (tafamidis) at preserving heart health among people with transthyretin amyloid cardiomyopathy (ATTR-CM), according to a new analysis of health insurance data in the U.S.
While both oral therapies are approved in the country to treat the rare heart disease, no clinical trials have tested the two ATTR-CM medications against each other. Now, a U.S. research team turned to claims data to “[evaluate] the real-world comparative effectiveness of [Attruby] versus [Vyndamax] in newly treated patients with ATTR-CM,” per the scientists.
The analysis found a “significantly lower risk” of heart-related events and death with Attruby compared with Vyndamax.
“The progressive nature of ATTR-CM is evident in all [previous] Phase 3 trials and is seen again here,” Richard Wright, MD, the study’s first author at the Pacific Heart Institute in Los Angeles, said in a press release from Bridgebio, the company that sells Attruby in the U.S. “This demonstrates the need for clinicians to proactively mitigate disease progression, and these data have the potential to meaningfully inform treatment selection in newly diagnosed patients and in patients currently on other treatments.”
The study’s researchers noted, however, that the findings should be interpreted with caution due to the natural limitations of health insurance data and the short follow-up time, which spanned a maximum of about eight months.
“Additional evaluation with longer follow-up, larger [patient groups], and/or … clinical outcomes [monitored over time] is warranted,” the scientists wrote.
The study, “Comparative Effectiveness of TTR Stabilizers for the Treatment of ATTR-CM Using Real-World Evidence,” was published in the journal Cardiology and Therapy. The work was funded by Bridgebio, and one of the study’s eight authors is an employee of the California-based company; three others work for Genesis Research Group, a consulting services firm.
In ATTR-CM, the protein transthyretin (TTR) forms toxic clumps that damage heart tissue, ultimately leading to heart failure. When the heart cannot pump blood efficiently, fluids begin to build up in the body, and patients need diuretic treatment, which is used to remove excess fluid and swelling caused by heart failure and other conditions.
Analysis finds 43% lower risk of heart-related events with Attruby
Attruby and Vyndamax are both oral TTR stabilizers that help prevent the protein from forming these clumps, or aggregates. Until late 2024, when Attruby was approved in the U.S., Vyndamax — which won approval in 2019 — was the only therapy cleared in the country for use by ATTR-CM patients.
Both treatments are indicated for reducing cardiovascular-related hospitalization or death in adults with ATTR-CM.
Clinical trials leading to the approvals of both Attruby and Vyndamax compared each drug against a placebo, but no trial to date has compared these therapies directly.
“In the absence of head-to-head [appropriately-controlled] trials, real-world comparative effectiveness data are needed to inform clinical decision-making and optimize patient outcomes,” the researchers wrote. With this in mind, the team used insurance data to compare outcomes between the two therapies among adults with ATTR-CM who had received no prior treatment.
Their analysis covered 170 patients who started on Attruby in late 2024 or early 2025, and 448 patients who began using Vyndamax in the same timeframe. All patients were selected using propensity score matching, a statistical technique used to ensure that the two groups were matched on as many pretreatment characteristics as possible.
About 1 in 5 participants were women, and about 1 in 4 were Black individuals, per the data. The team noted that “this real-world dataset includes higher proportions of under-represented populations, notably Black and female patients, than most ATTR-CM trials.”
Over a mean follow-up time of about 4.5 months and a maximum of about eight months, the researchers compared rates of diuretic intensification (DI), defined as the initiation of diuretic medications or an increase in their dose.
The significant reduction [of the need for diuretics], an early indicator of worsening heart failure, points to improved disease control and clinical stability [with Attruby].
The results showed that about 1 in 10 patients (12%) on Attruby experienced DI as did about 1 in 5 (21%) on Vyndamax. Mathematically, that works out to a 43% lower risk of DI with Attruby compared with Vyndamax, according to the team. Rates of DI for each group “separated within weeks of treatment initiation and remained divergent over time,” the researchers wrote.
According to Wright, “these findings further position [Attruby] as a differentiated TTR stabilizer in contemporary clinical practice.” The investigator added: “The significant reduction in diuretic intensification, an early indicator of worsening heart failure, points to improved disease control and clinical stability” with Attruby.
The researchers also assessed the risk of a composite measure that included DI alongside heart failure-related hospitalization and death of any cause. This similarly showed that the risk of the composite outcome was significantly lower with Attruby, by 34%, with a difference evident within weeks of treatment initiation.
Findings limited by use of insurance records, short follow-up time
The scientists noted that their analysis is limited by its reliance on insurance records, which are at best an imperfect surrogate for clinical data. Additionally, this study had a fairly short follow-up time. Thus, the researchers stressed a need for future studies to more comprehensively compare long-term outcomes between the two ATTR-CM drugs.
Overall, however, the data showed Attruby led to better outcomes than Vyndamax among U.S. patients, the researchers concluded.
“In this first real-world comparative effectiveness study, acoramidis demonstrated a significantly lower risk of DI and a clinical composite of DI, [heart failure-related hospitalization], and mortality compared with [Vyndamax],” the team wrote.
Still, “this study is limited by missing clinical information not typically captured or able to be measured from claims data,” the researchers wrote. The team also noted a “lack of [confirmation] of [heart-related] events, and limited follow-up time (maximum of [about] 8 months).” As such, they added, further study is needed.
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