Acoramidis, marketed as Attruby, represents a significant new medication now available to patients. This oral therapy recently received approval from the U.S. Food and Drug Administration (FDA), marking an important step forward in treating a specific heart condition. The FDA’s decision means that a new treatment option has been rigorously evaluated and deemed safe and effective for its intended use.
Understanding Acoramidis and Transthyretin Amyloid Cardiomyopathy
Acoramidis is a medication designed to address transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive heart disorder. ATTR-CM is characterized by the accumulation of abnormal protein deposits, called amyloid fibrils, in the heart muscle. These deposits make the heart walls stiff and prevent the left ventricle from relaxing and filling with blood properly, leading to a form of heart failure.
The disease can be categorized into two main types: hereditary ATTR-CM (hATTR-CM) and wild-type ATTR-CM (wATTR-CM). Hereditary ATTR-CM is caused by a variant in the transthyretin (TTR) gene. Wild-type ATTR-CM develops without a genetic mutation and is typically associated with aging. Both forms of ATTR-CM lead to similar heart problems, and untreated, the condition has a poor prognosis, highlighting an unmet medical need for effective therapies.
Key Details of the FDA Approval
The FDA approved Attruby (acoramidis) on November 22, 2024, specifically for treating adults with wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM). This approval aims to reduce cardiovascular death and hospitalizations in these patients.
The approval was based on evidence from a multicenter, international, randomized, double-blind, placebo-controlled study known as ATTRibute-CM. This Phase 3 trial involved 611 adult patients with either wild-type or hereditary ATTR-CM and evaluated the drug’s efficacy and safety over 30 months. The primary endpoints of the study included all-cause mortality and the cumulative frequency of cardiovascular-related hospitalizations.
Results from the ATTRibute-CM trial showed that at 30 months, a higher percentage of patients receiving acoramidis were alive (81%) compared to those on placebo (74%). Additionally, patients treated with acoramidis experienced fewer cardiovascular-related hospitalizations, with a mean of 0.3 per year compared to 0.6 per year in the placebo group.
How Acoramidis Works and Its Role in Treatment
Acoramidis stabilizes the transthyretin (TTR) protein, thereby preventing its breakdown and amyloid deposit formation in the heart. The drug is an orally administered, near-complete (90% or greater) stabilizer of TTR. By stabilizing TTR, acoramidis helps preserve the protein’s native function as a transport protein for thyroxine and vitamin A, which can positively impact cardiovascular outcomes.
This mechanism of action directly addresses the underlying pathology of ATTR-CM, aiming to slow disease progression and improve patient outcomes. The drug’s role in the treatment landscape is to provide a new first-line option that offers TTR stabilization and improves clinical outcomes. Early data from studies suggest that acoramidis can reduce all-cause mortality and cardiovascular hospitalization as early as three months after starting therapy.
Acoramidis is administered orally, typically twice daily. Clinical trial data indicated that common side effects observed included mild abdominal pain and diarrhea. The introduction of acoramidis offers a new pathway for managing ATTR-CM, potentially making it a more manageable chronic cardiovascular condition.