Bayer receives MHRA authorisation for acoramidis as a new treatment of transthyretin amyloidosis in adult patients with cardiomyopathy

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the use of Beyonttra®▼(acoramidis) in the UK for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).¹ ATTR-CM is a progressive, fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure.⁴ Patients with ATTR-CM face an ongoing risk of disease progression caused by continued amyloid deposition in the heart.⁴ Beyonttra is an orally administered, selective small molecule that stabilises transthyretin (TTR).¹

“ATTR-CM is a progressive and debilitating disease that poses significant challenges not only for patients but also for the healthcare systems. The condition profoundly impacts patients’ quality of life. Symptoms attributable to amyloidosis are usually nonspecific, varied and associated with low awareness, frequently resulting in delayed or completely missed diagnosis⁵, which may lead to delayed treatment and a worse prognosis. In the absence of intervention, ATTR-CM causes progressive heart failure leading to increased hospitalisations and escalating healthcare costs and is ultimately

fatal,6-8” said Professor Julian Gillmore, Professor of Medicine, University College London (UCL) Centre for Amyloidosis, UK. “The UK authorisation of Beyonttra is welcome news for patients living with the condition. Physicians in the UK now have another treatment option to slow the progression of symptoms and improve outcomes for patients with ATTR-CM.”

ATTR-CM has two forms: wild-type ATTR-CM and hereditary or variant ATTR-CM.⁹ Wild-type ATTR-CM generally affects older adults, particularly men, with a median survival of approximately 3.6 years if untreated.⁹ Hereditary or variant ATTR-CM is caused by inherited mutations in the TTR gene, resulting in less stable variants that may form amyloid fibrils⁹, with median survival ranging from approximately 2.5 to 3.4 years depending on the specific gene mutation without treatment.^9,10

Currently, estimating the prevalence of ATTR-CM in the UK is challenging due to potential under-diagnosis and under-reporting of the condition;¹¹ however, the number of new diagnoses made each year is increasing, partly due to increased utilisation of imaging techniques and the broader availability of non-invasive diagnostic tests.³ In 2024, a study using data from the UK Biobank suggested that approximately one in 1,000 people in the UK possess genetic variants likely linked to cardiac transthyretin (ATTR) amyloidosis.¹²

"We are pleased to bring Beyonttra to eligible patients in the UK. ATTR-CM is a challenging condition to manage, often leading to serious complications. The UK marketing authorisation of Beyonttra represents an important advancement for patients with ATTR-CM, offering an additional treatment option for healthcare professionals to manage this complex disease,” said Dr Joep Hufman, Country Medical Director, Bayer UK & Ireland. “As a next step, we are committed to continuing collaborating with health technology assessment bodies and healthcare professionals to ensure that eligible patients have timely access to this therapy, which may help improve outcomes by reducing their risk of cardiovascular events and slowing disease progression.”

Acoramidis, developed by BridgeBio Pharma, Inc. (Nasdaq: BBIO), was approved in the European Union in February 2025¹³ and in the U.S. by the Food and Drug Administration (FDA) under the brand name Attruby™ in November 2024¹⁴. BridgeBio holds the marketing rights for acoramidis in the U.S., while Bayer holds the exclusive marketing rights for the product in the European Union, the UK and Switzerland.

The marketing authorisation of Beyonttra in the UK is based on the pivotal results of the Phase III ATTRibute-CM study.¹ The Phase III trial was a multicentre, international, randomised, double-blind, placebo-controlled clinical study that investigated the efficacy and safety of acoramidis (compared to placebo), conducted in 632 adult participants (421 to the acoramidis group and 211 to the placebo group) with wild-type or variant (hereditary or de novo) ATTR-CM and heart failure NYHA (New York Heart Association) Class I-III, with current or prior symptoms of heart failure.¹ In the study, acoramidis was superior to placebo in the four-step primary hierarchical analysis with respect to death from any cause, cardiovascular-related hospitalisations (CVH), NT-proBNP (N-terminal pro-B-type natriuretic peptide), and 6-minute walk distance.² Acoramidis was generally well-tolerated. The overall incidence of adverse events was similar in the acoramidis group and the placebo group (98.1% and 97.6%, respectively).² The overall incidence of adverse events was similar in the acoramidis group and the placebo group (98.1% and 97.6%, respectively).² The most frequently reported adverse reactions were diarrhoea (11.6%) and gout (11.2%).¹

ENDS

About ATTR-CM

Transthyretin amyloid cardiomyopathy (ATTR-CM) is progressive and can be a fatal disease in adults, characterised by the deposition of abnormal protein in the heart as well as other organs and tissues. It occurs when a tetrameric protein called transthyretin (TTR) becomes unstable, caused by an inherited mutation in the TTR gene or due to aging, and dissociates into monomers. These monomers can misfold, aggregate, and form amyloid fibrils that deposit in various tissues and organs, including the heart muscle, leading eventually to heart failure. The disease is often diagnosed late, when the accumulation of amyloid in the heart has already occurred, and patients are symptomatic.⁷

About Acoramidis

Acoramidis is an orally administered, selective small molecule stabiliser of transthyretin (TTR). Acoramidis was designed to mimic the structure of a naturally occurring “mutation” of the TTR gene (T119M) that is associated with transthyretin protein that is more stable than wild-type transthyretin.²

About Bayer’s Commitment in Cardiovascular Diseases

Bayer is a leader in cardiology and is advancing a portfolio of innovative treatments in cardiovascular (CV) diseases of high unmet medical need. The strategy is to transform its portfolio into precision cardiology to support patients living with CVD, addressing the high CV disease burden as well as driving the long-term growth. Bayer’s portfolio already includes several investigational products and compounds in various stages of preclinical and clinical development.

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to www.bayer.co.uk

RP-BEY-GB-0011 / April 2025