The European Commission (EC) has granted marketing authorisation for Beyonttra (acoramidis), a treatment developed by Nasdaq-listed BridgeBio Pharma for wild-type or variant transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in adults.
This milestone allows Bayer, BridgeBio Pharma’s commercial partner in Europe, to oversee all marketing activities for the drug across the region.
Beyonttra is an orally administered, selective small molecule that stabilises transthyretin (TTR) with near-complete (≥90%) efficiency. ATTR-CM, a life-threatening condition, leads to restrictive cardiomyopathy and heart failure due to protein misfolding and deposition in cardiac tissues.
The European approval is based on findings from the ATTRibute-CM Phase 3 clinical trial, which evaluated acoramidis in 632 patients with symptomatic ATTR-CM. Participants were randomly assigned in a 2:1 ratio to receive either acoramidis or a placebo over 30 months.
The study demonstrated significant benefits, including reduced cardiovascular-related hospitalisations, improved survival rates, and better preservation of patients’ functional capacity and quality of life.
Moreover, time to the first clinical event (either all-cause mortality or cardiovascular-related hospitalisation) showed durable separation from placebo as early as three months into treatment.
Following this authorisation, Bayer plans to launch Beyonttra in the European Union (EU) in the first half of this year.
This regulatory approval triggers a $75m milestone payment from Bayer to BridgeBio Pharma under the terms of their licensing agreement, established in March 2024. BridgeBio Pharma will also receive tiered royalties starting in the low-thirties percentage on sales generated within the EU.
BridgeBio Pharma cardiorenal chief medical officer Jonathan Fox said: “The EU approval of acoramidis is a significant advancement for patients living with ATTR-CM in need of new disease-modifying treatments for their condition.
“This approval would not have been possible without the commitment of the clinical trial participants and their families, and the dedicated support of the physicians and scientists involved in the clinical program. Alongside our able partners at Bayer we look forward to this new opportunity to serve ATTR-CM patients across the European Union.”
Acoramidis has already gained approval in the US under the brand name Attruby, with the US Food and Drug Administration (FDA) granting marketing authorisation in November 2024.
The drug is currently under review by regulatory authorities in Japan and Brazil. These applications are supported by data from the ATTRibute-CM study, which highlighted acoramidis’s rapid clinical benefits.
Key outcomes from the trial include a 42% reduction in the composite of all-cause mortality and recurrent cardiovascular-related hospitalisation events at 30 months, alongside a 50% decrease in the cumulative frequency of cardiovascular-related hospitalisations compared to placebo.
Acoramidis is presently the only approved therapy for ATTR-CM in both the US and EU with a label specifying near-complete TTR stabilisation.
Bayer global product strategy and commercialisation executive vice president Christine Roth said: “Heart failure remains one of the most pressing challenges in global healthcare, with conditions like ATTR-CM often an undiagnosed cause.
“The EU approval of Beyonttra marks an important milestone bringing renewed optimism for patients living with ATTR-CM and offers physicians an additional fast-acting treatment option to protect these vulnerable patients by reducing their risk of cardiovascular events and slowing disease progression.”
