TTR stabilizer for transthyretin amyloidosis (ATTR)

Acoramidis is an investigational, oral drug designed to potently stabilize transthyretin (TTR). In 2023, BridgeBio announced positive results from ATTRibute-CM, a Phase 3 study of acoramidis in ATTR cardiomyopathy (ATTR-CM). For patients with ATTR-CM, TTR stabilization offers the chance to both preserve the cardio-protective benefits of TTR and address the root cause of disease. The U.S. FDA has accepted the New Drug Application (NDA) and the European Medicines Agency (EMA) accepted the Marketing Authorization Application with additional global regulatory submissions planned.

The safety and efficacy of acoramidis have not been fully established. There is no guarantee that acoramidis will receive health authority approval or become commercially available in any country for the uses being investigated.

acoramidis facts



genetic source

TTR (transthyretin)

clinical phase

Phase 3


small molecule

our approach

Acoramidis is a next-generation TTR stabilizer designed by researchers at Stanford University to mimic the structure and stabilizing effect of the naturally protective T119M variant. T119M is known to protect individuals from developing ATTR-CM due to the formation of hydrogen bonds at the center of the molecule that stabilize the protein approximately 40-fold beyond the native structure.1

In an analysis of an open-label extension of a Phase 3 study of TTR-stabilizing medicine in patients with ATTR-CM, it was found that a greater level of TTR stabilization led to improved clinical benefit in ATTR-CM patients.2

presentations & publications

ISA 2024 – Early increase in serum transthyretin level is an independent predictor of improved survival in ATTR cardiomyopathy 05.29.2024
ISA 2024 – Acoramidis treatment-related increase in serum TTR is associated with lower cardiovascular mortality in ATTR-CM 05.29.2024
ISA 2024 – Acoramidis treatment-related increase in serum TTR is associated with a lower risk of cardiovascular hospitalization in ATTR-CM patients 05.29.2024
ISA 2024 – Acoramidis achieves early reduction in cardiovascular death or hospitalization in transthyretin amyloid cardiomyopathy (ATTR-CM) 05.29.2024 05.29.2024
ISA 2024 – Rationale & design of ACT-EARLY 05.29.2024
ESC Heart Failure 2024 – ITT Sensitivity Analysis and Sub-Analysis Comparing Acoramidis and Placebo in Stage 4 CKD in ATTRibute-CM 05.11.2024
ESC Heart Failure 2024 – Improved Health-Related Quality of Life in Acoramidis-Treated Patients with ATTR-CM, Demonstrated by Improvements in KCCQ Scores 05.13.2024
ESC Heart Failure 2024 – EQ-5D Analysis from the ATTRibute-CM Study 05.13.2024
ESC Heart Failure 2024 – NT-proBNP Insights from the ATTRibute-CM Study
Acoramidis – ACC 2024 – CMR Imaging Substudy from ATTRibute-CM Phase 3 Study 04.07.2024
The New England Journal of Medicine, Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy 01.10.2024
Acoramidis – AHA 2023 – Clinical Outcome Improvements 11.12.2023
acoramidis – hfsa 2023 – 4 year update from ole phase 2 study 10.08.2023
ATTRibute-CM Detailed Results From ESC Congress 2023 08.28.2023
Acoramidis – ESC 2023 – ATTRibute-CM-Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy 08.27.2023
Acoramidis – ESC 2023 – Acoramidis Produces Near-Complete TTR Stabilization in Variant ATTR Patients that is Greater than that Achieved with Tafamidis 08.26.2023
ATTRibute-CM Phase 3 Topline Results 07.17.2023
acoramidis – isa 2022 – updated analysis ongoing phase 2 ole 08.12.2022
acoramidis – acc 2022 – updated analysis ongoing phase 2 ole 04.02.2022
acoramidis – acc 2022 – unmet needs among patients with attr 04.01.2022
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  1. Hammarstrom, P. et al. PNAS 2002, 99:16427 – 16432.
  2. Judge et al. Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy. JAMA Cardiol. 2019;74(3)285-295.