Acoramidis Approved in Europe for ATTR Cardiomyopathy

Acoramidis, a transthyretin stabilizer, has been approved by European regulators for the treatment of wild-type or variant transthyretin amyloidosis in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), according to an announcement from manufacturer BridgeBio today.

The approval follows a positive opinion from the Committee for Medicinal Products for Human Use in 2024. The drug, marketed as Attruby in the US, joins tafamidis (Vyndaqel and Vyndamax; Pfizer) as the second therapy approved in Europe for adults with ATTR-CM. The US Food and Drug Administration approved acoramidis in November 2024 and tafamidis in 2019. In Europe, acoramidis will be marketed as Beyonttra.

Approval from the European Commission is based on results of the ATTRibute-CM trial. As reported by TCTMD, acoramidis led to a significantly better primary composite endpoint that included mortality, morbidity, and walking ability at 30 months when compared with placebo.

Unlike in ATTR-ACT with tafamidis, acoramidis did not reduce the risk of all-cause mortality, but experts chalked this up to trial patients not being as sick in ATTRibute-CM. Researchers have previously noted that there’s been a change in the ATTR-CM population since the earlier studies, as patients are now diagnosed earlier and with less symptom burden so the lack of effect on all-cause mortality alone wasn’t surprising.

The treatments for ATTR-CM are famously expensive, with Pfizer first drawing the ire of physicians for listing the drug at roughly $225,000 per year. In the US, acoramidis is priced at approximately $244,000 annually. There’s been no word on what the drug will cost in Europe.

Another agent in development for the treatment of ATTR-CM is the small-interfering RNA vutrisiran (Amvuttra; Alnylam). Unlike tafamidis and acoramidis that stabilize the malformed TTR protein, vutrisiran is a gene silencer of the defective TTR mRNA.