The United Kingdom Medicines and Healthcare Products Regulatory Agency has approved acoramidis for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM), BridgeBio announced in a recent press release.
Acoramidis, marketed in Europe as Beyonttra®, is a transthyretin (TTR) stabilizer, meaning it prevents TTR from breaking down and forming amyloid plaques in the heart.
The drug, which is administered orally, will be available to adults who have both wild-type ATTR-CM, which has no known genetic cause, and hereditary ATTR-CM, which has a known genetic basis.
“Acoramidis is the first and only approved ATTR-CM treatment in the [United States, European Union, United Kingdom] and Japan that all have a label specifying near-complete stabilization (≥90%),” of TTR, the company wrote. This approval comes just five months after the United States Food and Drug Administration approved acoramidis (marketed in the U.S. as Attruby™) for patients with ATTR-CM in November 2024.
Read more about ATTR-CM therapies
Approval was based on the results of the ATTRibute-CM clinical trial, which showed the fastest response seen to date in a Phase 3 study of patients with ATTR-CM. Within three months, the time to all-cause mortality or cardiovascular-related hospitalization was substantially reduced in patients receiving acoramidis compared to those receiving a placebo.
Furthermore, at Month 30, the sum of all cardiovascular hospitalizations was reduced by 50% in patients receiving acoramidis compared to patients receiving a placebo. Similarly, patients taking acoramidis experienced a 42% reduction in the combined risk of all-cause mortality and recurring cardiovascular hospitalizations at Month 30.
The most common adverse reactions reported were diarrhea and upper abdominal pain, but most of these reactions were mild. Only 9.3% of patients taking acoramidis discontinued the drug due to side effects, compared to 8.5% of patients taking a placebo, indicating similar rates of discontinuation.
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