Bayer receives MHRA authorisation for Beyonttra®▼ (acoramidis) as a new treatment of transthyretin amyloidosis in adult patients with cardiomyopathy

• Beyonttra® (acoramidis), an orally administered, selective small molecule, is indicated for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM)¹
• The UK marketing authorisation is based on the results of the Phase III ATTRibute-CM study investigating the efficacy and safety of acoramidis in adult patients with ATTR-CM²
• ATTR-CM is a progressive and fatal condition that requires early diagnosis, management, and specific treatment³

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

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 Phase III ATTRibute-CM 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 in the study.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

“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 diagnosis,⁵ 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 eligible 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.”

"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.”

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

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.

ENDS

Bayer Media Contact:

Veronica Yao, +44 (0) 7870 485 926 Email: veronica.yao@bayer.com

Notes to Editors

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.⁸

About Acoramidis and the Phase III study results

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

In the ATTRibute-CM Phase III study, acoramidis met the primary efficacy endpoint. The primary objective of the ATTRibute-CM Phase III study was to establish superiority of acoramidis versus placebo on a hierarchical endpoint that included all-cause mortality (ACM), cumulative frequency of cardiovascular-related hospitalisations (CVH), change from baseline in NT-proBNP and 6-minute walk distance at month 30.²

A total of 632 patients underwent randomisation (421 to the acoramidis group and 211 to the placebo group) in the study. When compared to placebo, acoramidis 800mg twice daily demonstrated statistically significant clinical benefit on the primary hierarchical endpoint of all-cause mortality, cardiovascular hospitalisation, change from baseline in NT-proBNP, 6-minute walk distance in adults with ATTR-CM through month 30 (p<0.001)². Key secondary outcomes included the change from baseline until month 30 in the 6-minute walk distance and KCCQ-OS (Kansas City Cardiomyopathy Questionnaire – Overall Summary) score.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 most frequently reported adverse reactions were diarrhoea (11.6%) and gout (11.2%).¹

Patients enrolled in the ATTRibute-CM study could enter an Open-Label Extension (OLE) study after 30 months. The OLE data for continuous acoramidis demonstrated a relative risk reduction in ACM of 33.7% (HR: 0.64, CI 0.47-0.88, p-value=0.006) by 42 months, highlighting its potential to improve long-term survival outcomes compared to placebo-to-acoramidis. No additional safety signals have been reported during the interim analysis of the OLE at month 42.

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.

Forward-Looking Statements 

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.co.uk. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments. 

References: 

1. Summary of Product Characteristics (SmPC) for Beyonttra 356 mg film-coated tablets. 
2. Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, Grogan M, Hanna M, Hoffman J, Masri A, Maurer MS. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. New England Journal of Medicine. 2024 Jan 11;390(2):132-42.
3. Dulce Brito, et al. World Health Federation Consensus on Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM). Glob Heart. 2023 Oct 26;18(1):59. doi: 10.5334/gh.1262
4. Gillmore JD, Rezk T. Amyloid heart disease module 2: management. The British Journal of Cardiology. April 2021doi:10.5837/bjc.2021.016 
5. Rintell et al. Orphanet J Rare Dis. (2021) 16:70. https://doi.org/10.1186/s13023-021-01706-7 

6. Rozenbaum MH, et al. Impact of delayed diagnosis and misdiagnosis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM): a targeted literature review. Cardiology and therapy. 2021;10:141-59.

7. Mallus MT and Rizzello V. Treatment of amyloidosis: present and future. 2023;21;25(Suppl B):B99-B103.

8. Jain A, Zahra F. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Updated 27 April 2023. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK574531/  Last accessed: March 2025.

9. Frederick L. Ruberg, MD, et al. Transthyretin Amyloid Cardiomyopathy: JACC State-of-the-Art Review. J Am Coll Cardiol. 2019 June 11; 73(22): 2872–2891. doi:10.1016/j.jacc.2019.04.003  
10. Sattianayagam PT, Hahn AF, Whelan CJ et al. Cardiac phenotype and clinical outcome of familial amyloid polyneuropathy associated with transthyretin alanine 60 variant. European Heart Journal. 2012;33(9):1120-1127. 
11. Patel K, Hawkins P. Cardiac amyloidosis: where are we today? Journal of internal medicine. 2015;278(2):126‐44. 
12. Nay Aung, et al. Prevalence, Cardiac Phenotype, and Outcomes of Transthyretin Variants in the UK Biobank Population. JAMA Cardiol. 2024;9(11):964-972. doi:10.1001/jamacardio.2024.2190. 
13. Beyonttra. European Commission Decision. European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/EPAR/beyonttra. Last accessed: March 2025.
14. BridgeBio press release. Attruby™ (acoramidis), a Near Complete TTR Stabilizer (≥90%), approved by FDA to Reduce Cardiovascular Death and Cardiovascular-related Hospitalization in ATTR-CM Patients. Last accessed: March 2025.

RP-BEY-GB-0010 / April 2025