Beyonttra
Authorised
This medicine is authorised for use in the European Union
acoramidis
MedicineHumanAuthorised
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
Beyonttra is a medicine used in adults to treat cardiomyopathy (damage to the heart muscle) caused by variant or wild-type transthyretin amyloidosis (ATTR-CM), a disease in which abnormal transthyretin (TTR) proteins form clumps called amyloids, which build up mainly in the heart.
Variant transthyretin amyloidosis occurs when there is a mutation (change) in the gene for the TTR protein, causing it to misfold and form amyloids. In wild-type transthyretin amyloidosis there is no gene mutation, but the TTR protein still misfolds.
Beyonttra contains the active substance acoramidis.
Beyonttra can only be obtained with a prescription and treatment should be started by a doctor experienced in the management of patients with ATTR-CM. The medicine is available as tablets that are taken by mouth twice daily.
For more information about using Beyonttra, see the package leaflet or contact your doctor or pharmacist.
In ATTR-CM, the TTR protein is unstable and breaks apart easily into single units. These single units misfold and clump together forming amyloid deposits in the heart, causing stiffening of the heart and making it harder for it to pump blood.
The active substance in Beyonttra, acoramidis, stabilises the TTR protein by forming strong connections with certain parts of the protein. This helps prevent the TTR protein from breaking apart, which slows down the progression of ATTR-CM.
In a main study involving 632 patients with either variant or wild-type ATTR-CM with symptoms of heart failure, Beyonttra was shown to be more effective than placebo (a dummy treatment) at slowing down the heart damage caused by the disease.
The main measure of effectiveness was a combination of four different outcomes. These included the overall death rate, the number of hospitalisations due to issues related to the heart or blood circulation, the change in levels of NT-proBNP (a hormone released by the heart when it is under stress; levels increase during heart failure), as well as the change in the distance patients could walk in 6 minutes. Beyonttra was compared with placebo using a method that evaluated the win ratio based on these four different outcomes. A higher win ratio means that a treatment has a greater benefit than another.
After 30 months of treatment, the win ratio was around 1.77, meaning those given Beyonttra had around a 77% higher chance of experiencing a benefit with Beyonttra than those given placebo. The overall death rate was 19.3% for those given Beyonttra compared with 25.7% for those given placebo, and the distance patients could walk in 6 minutes was on average 366 metres with Beyonttra compared with 322 metres on average with placebo.
For the full list of side effects and restrictions with Beyonttra, see the package leaflet.
The most common side effects with Beyonttra (which may affect more than 1 in 10 people) include diarrhoea and gout (high levels of uric acid in the blood causing symptoms especially painful inflammation in the joints).
ATTR-CM is a life-threatening disease that progresses rapidly. At the time of approval of Beyonttra, there were limited treatment options for patients with the disease. In the main study, Beyonttra was shown to be more effective than placebo at preventing death due to ATTR-CM. However, this effect was less pronounced in patients with more advanced symptoms of heart failure. Additionally, patients given the medicine walked around 40 metres further in 6 minutes than those given placebo. The main side effects seen with Beyonttra were related to the stomach and gut, such as diarrhoea, and gout.
The European Medicines Agency therefore decided that Beyonttra’s benefits are greater than its risks and that it can be authorised for use in the EU.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Beyonttra have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Beyonttra are continuously monitored. Suspected side effects reported with Beyonttra are carefully evaluated and any necessary action taken to protect patients.
Beyonttra received a marketing authorisation valid throughout the EU on 10 February 2025.
Product information
10/02/2025
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Beyonttra
- Active substance
- acoramidis hydrochloride
- International non-proprietary name (INN) or common name
- acoramidis
- Therapeutic area (MeSH)
- Amyloid Neuropathies, Familial
- Anatomical therapeutic chemical (ATC) code
- C01EB25
Pharmacotherapeutic group
Cardiac therapyTherapeutic indication
For the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
News on Beyonttra
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