The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending acoramidis for marketing authorization in the European Union (EU). Acoramidis is a selective, orally administered small molecule designed to stabilize transthyretin (TTR) and is indicated for the treatment of adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive and often fatal heart condition that manifests as infiltrative, restrictive cardiomyopathy, leading to heart failure. In laboratory studies, acoramidis showed near-complete stabilization of TTR, and in the Phase III ATTRibute-CM trial, the compound demonstrated significant benefits on cardiovascular outcomes.
This positive CHMP opinion is based on the pivotal results from the ATTRibute-CM study, which evaluated the efficacy and safety of acoramidis administered twice daily compared to a placebo in patients with ATTR-CM. The study found that acoramidis significantly reduced the composite of all-cause mortality (ACM) and cardiovascular-related hospitalizations (CVH), showing clear superiority over placebo.
“ATTR-CM is frequently misdiagnosed or recognized too late, which can worsen patient outcomes. The condition progresses relentlessly without treatment and is ultimately fatal,” said Professor Julian Gillmore, a leading expert in amyloidosis at University College London (UCL). “The CHMP’s positive opinion offers hope for patients living with this condition. The prospect of a treatment that nearly completely stabilizes TTR provides a new opportunity to slow the progression of symptoms and improve outcomes for ATTR-CM patients.”
Christine Roth, Executive Vice President of Global Product Strategy and Commercialization at Bayer, emphasized the urgent need for innovative treatments for ATTR-CM. “Patients with ATTR-CM often experience a significant decline in quality of life due to the physical and functional challenges associated with the disease. The positive CHMP recommendation is a major milestone in addressing this life-threatening heart condition. We are eager to receive the European Medicines Agency’s approval for acoramidis, which is expected in early 2025, and to make this new treatment available to patients as quickly as possible.”
The final decision from the European Commission on the marketing authorization is anticipated in the coming months. Acoramidis, developed by BridgeBio Pharma, Inc. (Nasdaq: BBIO), was recently approved by the U.S. Food and Drug Administration (FDA), with a label specifying near-complete stabilization of TTR. While BridgeBio holds the marketing rights for acoramidis in the U.S., Bayer has exclusive marketing rights for the product in Europe. Pending regulatory approval, Bayer plans to launch acoramidis in Europe in the first half of 2025.
Bayer and BridgeBio have been collaborating on the development of acoramidis since March 2024, combining Bayer’s expertise in cardiovascular disease and European cardiovascular infrastructure with BridgeBio’s leadership in the emerging field of ATTR-CM. This partnership aims to bring innovative treatments to patients in Europe, addressing a significant unmet need in the management of ATTR-CM.
Acoramidis represents a significant advancement in the treatment of transthyretin amyloid cardiomyopathy, a disease that currently has limited treatment options. The near-complete stabilization of TTR demonstrated by acoramidis offers the potential to slow disease progression, improve patient outcomes, and ultimately extend survival in a patient population that has long been in need of more effective therapies. With the upcoming European approval, acoramidis could become a cornerstone in the management of ATTR-CM, providing hope for many patients affected by this life-threatening condition.
