New ATTR-CM drug linked to positive long-term data as FDA nears final decision
Transthyretin amyloid cardiomyopathy (ATTR-CM) patients treated with the amyloidosis drug acoramidis are associated with significantly lower mortality and hospitalization rates, according to new 42-month data from the ATTRibute-CM phase 3 trial. Acoramidis is currently being reviewed by the U.S. Food and Drug Administration (FDA), and a potential approval could be announced soon. Such an approval would bring an additional treatment to the market for cardiac amyloidosis patients.
The data were presented as a late-breaking trial during the American Heart Association (AHA) 2024 Scientific Sessions conference in Chicago by Daniel Judge, MD. Judge is the director of the cardiovascular genetics program, the Edwin W. and Teresa H. Rogers Endowed Chair for Cardiovascular Research and a professor of medicine at the Medical University of South Carolina. He spoke about the findings in an interview with Cardiovascular Business.
"Previously, we had shown that all-cause mortality, cardiovascular hospitalization and six-minute walk test distance was better. We reached statistical significance from the prior work, but that wasn't enough for some of the people who were looking for a clear mortality benefit in a disease that causes so much death. And now as we extend the study, we do see a mortality benefit that's very clear," Judge explained.
Many ATTR-CM patients are treated by heart failure cardiologists, and Judge said the key thing they want to know is how often people are hospitalized because of the condition. There is hope that the drug can help reduce such admissions. He said previous data showed the impact on hospitalizations was significant, but the new data show things get better the longer a patient is on the drug, underscoring the progressive nature of ATTR-CM, which involves transthyretin protein deposits damaging the heart more as time goes on.
"The drug works, and that's really good news. But one of the big signals is, the earlier you start treatment, the better they get. And the longer you wait ... the harder it is to see that type of a benefit," Judge exaplained.
The initial study included 421 patients on acoramidis vs. 211 on placebo. The ATTRibute-CM open-label extension (OLE) study included 263 patients on acoramidis compared to 226 on placebo
Judge said acoramidis is a selective TTR stabilizer for the treatment of patients with ATTR-CM that achieves near-complete TTR stabilization in more than 90% of patients.
The study found a relative risk reduction of 33.7% in all cardiac mortality. The risk of first cardiovascular hospitalization has a relative risk reduction of 41%. At 42 months, the relative risk reduction of combined cardiac mortality and cardiovascular hospitalizations was 48%.
Expanding cardiac amyloidosis treatment options
ATTR-CM has seen growing interest since 2019, when the first FDA-approved treatment, tafamidis, became available. Acoramidis, an oral transthyretin stabilizer, offers a new option for both wild-type and hereditary forms of the disease. Judge explained that having more therapies available could improve patient outcomes and give providers greater flexibility.
“We are diagnosing patients earlier, and their outcomes are improving,” he said, adding that biomarkers like N-terminal pro-B-type natriuretic peptide (NT-proBNP) and prealbumin levels can guide treatment response. Elevated prealbumin levels, for example, indicate effective protein stabilization.
The field of amyloidosis treatment is rapidly evolving. Noninvasive diagnostic tools and expanding therapeutic options are transforming patient care, allowing for earlier intervention and better management of this once-untreatable condition.
Dave Fornell has covered healthcare for more than 17 years, with a focus in cardiology and radiology. Fornell is a 5-time winner of a Jesse H. Neal Award, the most prestigious editorial honors in the field of specialized journalism. The wins included best technical content, best use of social media and best COVID-19 coverage. Fornell was also a three-time Neal finalist for best range of work by a single author. He produces more than 100 editorial videos each year, most of them interviews with key opinion leaders in medicine. He also writes technical articles, covers key trends, conducts video hospital site visits, and is very involved with social media. E-mail: dfornell@innovatehealthcare.com