First-of-a-kind EU approval granted for oral small molecule

The European Commission has approved acoramidis as the first near complete TTR stabiliser (≥90 percent) to treat transthyretin amyloidosis in adults with cardiomyopathy (ATTR-CM) patients in the US and EU.

BEYONTTRA™ (acoramidis) is indicated for adults with wild-type or variant ATTR-CM. This disease is progressively fatal and results in heart failure, BridgeBio Pharma, Inc. explained.

“[Acoramidis] has demonstrated improved benefit on all-cause mortality and cardiovascular-related hospitalisations in as few as three months, a very important accomplishment for patients. We are pleased that people living with ATTR-CM will have access to another treatment option in the EU,” commented Dr Marianna Fontana, PhD, Professor of Cardiology and Honorary Consultant Cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London.

Data on efficacy of acoramidis in ATTR-CM

The Commission’s approval is based on findings from the pivotal ATTRibute-CM Phase III study of acoramidis. This trial demonstrated the most rapid benefit seen in any Phase III study of ATTR-CM to date, according to BridgeBio.

Key results include that the company’s oral small molecule medicine enabled significant reduction of cardiovascular-related hospitalisation, improving survival, and preserving functional capacity at month 30, the data showed.

Further analysis reported that the more efficient stabilisation by acoramidis “was attributed to its primarily enthalpic binding mode (which involves hydrogen bonding mimicking the T119M variant) as compared with the predominantly entropic (hydrophobic) binding mode of tafamidis and diflunisal”.

The US Food and Drug Administration (FDA) granted approval of acoramidis as Attruby™ as a near complete TTR stabiliser in November 2024.

Under a collaboration agreed with Bayer in March 2024, BridgeBio will receive a $75 million upon EU approval of acoramidis. Bayer will be responsible for commercialisation for the medicine in the region, BridgeBio added.