The UK’s Medicines & Healthcare Products Regulatory agency has approved Acoramidis (Beyonttra) for the treatment of adult patients with cardiomyopathy caused by transthyretin amyloidosis (ATTR-CM), including both wild-type and hereditary (variant) forms.
In transthyretin amyloidosis-related cardiomyopathy (ATTR-CM), the transthyretin (TTR) protein becomes unstable and breaks apart, forming amyloid deposits that stiffen the heart muscle and impair its function. Acoramidis hydrochloride, the active ingredient in the newly approved treatment, helps slow disease progression by stabilizing the TTR protein and preventing the formation of these harmful amyloid clusters.
Acoramidis has undergone fast track approval through International Recognition Procedure (IRP) followed by approval by the European Medicines Agency (EMA). In order to speed up patient access to safe, effective & high-quality medicines.
The approval is supported by results from a large international clinical trial involving 632 patients with ATTR-CM. Participants receiving acoramidis showed significantly better outcomes compared to those given a placebo:
- 77% greater chance of overall clinical benefit
- Reduced cardiovascular-related hospitalizations
- Improved survival rates
- Better quality of life
- Stabilized TTR protein levels and reduced cardiac stress markers
- Improved walking distance in 6-minute walk tests
The most commonly reported side effects include diarrhea and gout (painful joint inflammation), affecting more than 1 in 10 users.
The recommended daily dose is two tablets. However, case should be monitored by an experienced doctor who has previous experience with ATTR-CM patients. And any side effects should be reported to doctor, nurse, pharmacist or directly to the yellow card scheme.
Learn more: Acoramidis approved to treat wild-type or variant transthyretin amyloidosis in adults with cardiomyopathy - GOV.UK
