BridgeBio wins ANVISA approval for acoramidis in ATTR-CM
BridgeBio Pharma has secured marketing authorisation from Brazil's ANVISA for acoramidis, branded locally as BEYONTTRA, to treat wild-type and variant transthyretin amyloid cardiomyopathy (ATTR-CM) in adults. The approval adds Brazil to a roster of major markets — including the United States, European Union, United Kingdom, Japan, and Switzerland — where the oral, small-molecule TTR stabiliser is now licensed, all with label language specifying near-complete (≥90%) stabilisation of the transthyretin protein.
The ANVISA decision is supported by data from the pivotal ATTRibute-CM Phase 3 trial, which enrolled 632 participants with symptomatic ATTR-CM and randomised them 2:1 to acoramidis or placebo over 30 months. The study met its primary clinical endpoints, with acoramidis delivering a 42% reduction in composite all-cause mortality and recurrent cardiovascular hospitalisation (CVH) events relative to placebo, and a 50% reduction in the cumulative frequency of CVH events. Notably, the company reports numerically fewer cumulative cardiovascular events were already apparent by Month 1 — a speed of benefit the company describes as the most rapid seen in any Phase 3 ATTR-CM study to date.
Clinical profile and safety
The drug's tolerability data are broadly reassuring. Diarrhoea was reported in 11.6% of acoramidis-treated patients versus 7.6% on placebo, and upper abdominal pain in 5.5% versus 1.4%; both adverse events were predominantly mild and rarely led to discontinuation. Discontinuation rates due to adverse events were comparable between arms — 9.3% versus 8.5% — a signal that will help prescribers counsel patients on adherence.
Fábio Fernandes, Director at the Heart Institute of the University of São Paulo Medical School and a trial investigator, described the results as "profoundly encouraging," noting that the ATTR-CM patient population in Brazil has historically faced delayed diagnosis and limited therapeutic options. Jonathan Fox, Chief Medical Officer of BridgeBio Cardiorenal, said the authorisation "brings new hope to communities where ATTR-CM is increasingly recognised," reflecting the company's stated ambition to pursue broad global access.
Market context and competitive landscape
ATTR-CM is a progressive, life-threatening disease caused by misfolded transthyretin protein depositing in cardiac tissue. Awareness of the condition has grown sharply over the past decade following the commercialisation of tafamidis (Pfizer's Vyndaqel and Vyndamax), which was the first approved TTR stabiliser and remains the dominant product in this class globally. BridgeBio is positioning acoramidis on the basis of its ≥90% stabilisation rate — a mechanistic differentiation point that was absent from tafamidis's original label and has featured prominently in BridgeBio's regulatory submissions worldwide.
Beyond small-molecule stabilisers, the ATTR-CM space is contested by RNA-interference therapies. Alnylam's patisiran and vutrisiran were initially developed for the hereditary polyneuropathy form of ATTR disease but have attracted regulatory and commercial interest across cardiac indications as well. The competitive dynamic between stabilisers and silencers is likely to intensify as longer-term outcomes data mature and payers in markets such as Brazil negotiate pricing frameworks.
BridgeBio has partnered with Biopas — a Swixx BioPharma subsidiary with an established Latin American commercialisation platform — to launch BEYONTTRA in Brazil, with commercial activities expected to begin in the second half of 2026. The choice of a specialist regional partner rather than a large multinational distributor signals a pragmatic market-entry strategy suited to Brazil's complex reimbursement environment through the Sistema Único de Saúde (SUS) and the supplementary health sector. Investors will be watching for reimbursement decisions and early prescription uptake figures as the key commercial milestones over the next twelve months.