Cardiac Amyloidosis

With the first drug treatments for cardiac amyloidosis recently entering the market, there has been an explosion of interest to diagnose and care for these patients. It is considered a rare disease, but many experts now say it is actually just be under diagnosed. The disease is caused by protein misfolding. Normally soluble proteins in the bloodstream become insoluble and deposit abnormally in the tissues and organs throughout the body. There are three main kinds of amyloid that affect the heart, light chain amyloid (AL) and two types of transthyretin amyloid (ATTR or TTR). The first type of ATTR is hereditary, or familial amyloid, and the second is wild type, or age-related TTR amyloid. Nuclear imaging, echocardiography, CT and MRI all play roles in diagnosing amyloid and in determining the subtype, which is required for targeted treatment. 

Cardiac amyloidosis ATTR-CM drug promotion at the 2024 American Heart Association meeting.

High cost of cardiac amyloidosis drugs may limit access for low-income patients

"We cannot accept a system where only those with means can benefit from life-changing drugs. We are a wealthy nation that spends trillions on healthcare. We must ensure that income and wealth do not determine access," explained JACC Editor-in-Chief Harlan M. Krumholz, MD.

Ultromics EchoGo Amyloidosis artificial intelligence

AI-enhanced echocardiography improves early detection of cardiac amyloidosis

The first FDA-cleared AI model of its kind was found to be an accurate screening tool for cardiac amyloidosis.

heart drugs with stethoscope

Work begins on first ATTR-CM study of its kind

ACT-EARLY is testing the hypothesis that treating asymptomatic carriers of a pathogenic TTR variant with the drug acoramidis could delay amyloidosis from developing.

Alnylam Pharmaceuticals has received U.S. Food and Drug Administration (FDA) approval for vutrisiran, a subcutaneous injection the company sells under the brand name Amvuttra, to treat adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM).

FDA approves vutrisiran to treat ATTR-CM

The subcutaneous drug, sold under the brand name Amvuttra, joins an ATTR-CM market that already includes Pfizer’s tafamidis (Vyndaqel, Vyndamax) and BridgeBio’s acoramidis (Attruby).

cardiac amyloidosis on bone scan

Continuation of 99mTc-PYP shortage prompts need for alternative cardiac amyloidosis imaging

After three years of intermittent shortages of nuclear imaging tracer technetium-99m pyrophosphate, there are no signs of the shortage abating.

pharmaceutical drug approval process

FDA approves acoramidis, BridgeBio’s new ATTR-CM drug

Acoramidis, a selective transthyretin stabilizer being sold under the brand name Attruby, is now the second ATTR-CM medication to gain FDA approval.

Daniel Judge, MD, director of the cardiovascular genetics program, Medical University of South Carolina, the Edwin W. and Teresa H. Rogers Endowed Chair for Cardiovascular Research, Medical University of South Carolina, presented the results of the late-breaking ATTRibute-CM study for the drug acoramidis used to treat transthyretin amyloidosis at AHA 2024.

ATTR-CM drug acoramidis, now approved by the FDA, linked to positive long-term data

Cardiovascular Business spoke with cardiologist Daniel Judge, MD, about the long-term benefits of acoramidis, a new drug therapy that just received FDA approval to treat transthyretin amyloid cardiomyopathy.

The rapid rise of artificial intelligence (AI) has helped cardiologists, radiologists, nurses and other healthcare providers embrace precision medicine in a way that ensures more heart patients are receiving personalized care.

FDA clears AI screening tool for cardiac amyloidosis

Ultromics designed EchoGo Amyloidosis to evaluate routine echocardiogram results for signs of cardiac amyloidosis. It received the FDA's breakthrough device designation back in 2023.