Acoramidis

Acoramidis is a selective stabilizer of transthyretin (TTR). Acoramidis binds TTR at thyroxine binding sites and slows dissociation of the TTR tetramer into its constituent monomers, the rate-limiting step in amyloidogenesis.

• ATTRUBY is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. ATTRUBY is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.

• None. None.contraindicated

The recommended dosage of ATTRUBY is 712 mg orally twice daily. ( 2.1 ) 2.1 Recommended Dosage The recommended dosage of ATTRUBY is 712 mg orally twice daily (with or without food). Swallow tablets whole; do not cut, crush, or chew.

6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The safety data reflect the exposure of 421 participants with ATTR-CM to ATTRUBY 712 mg (administered as two 356 mg tablets) administered orally twice daily in a randomized, double-blind, placebo-controlled trial of 30 months fixed treatment duration. The median duration of exposure to ATTRUBY in the safety population was 29 months. There was a higher frequency of gastrointestinal (GI) adverse reactions such as diarrhea 11.6% versus 7.6% and upper abdominal pain 5.5% versus 1.4% in the ATTRUBY versus placebo group, respectively. The majority of these GI adverse reactions were categorized as mild and resolved without drug discontinuation. A similar proportion of ATTRUBY-treated and placebo-treated participants discontinued study drug because of an adverse event (9.3% and 8.5%, respectively). Laboratory Tests Increase in Serum Creatinine and Decrease in eGFR Initiation of ATTRUBY causes an increase in serum creatinine and decrease in eGFR which generally occurs within 4 weeks of starting therapy and stabilizes. In a trial of adults with ATTR-CM, a mean increase in serum creatinine of 0.2 and 0.0 mg/dL and a mean decrease in eGFR of 8.2 and 0.7 mL/min/1.73 m 2 was observed in the ATTRUBY and placebo groups, respectively, at Day 28.

8.1 Pregnancy Risk Summary Available data with acoramidis use in pregnant women are insufficient to establish a drug associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. In animal reproductive studies in rats and rabbits, no embryofetal abnormalities were observed at exposures up to 34 times and 13 times the clinical exposure at the maximum recommended human dose, respectively ( see Data ). The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defects, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. Report pregnancies to the BridgeBio reporting line at 1-844-550-2246. Data Animal Data In pregnant rats, oral administration of acoramidis (0, 50, 350, and 1,000 mg/kg/day) throughout organogenesis did not result in any adverse effects on embryofetal development at up to 1,000 mg/kg/day, approximately 34 times the clinical exposure at the maximum recommended human dose (MRHD) based on AUC.

Metabolism

The systemic exposures (C max and AUC) increase in a less than dose proportional manner following single and multiple doses of acoramidis. Over the dose range from 89 mg twice daily to 712 mg twice daily, AUC increases only 130%.

There is no clinical experience with overdose. In case of suspected overdose, treatment should be symptomatic and supportive.

• Nov 22, 2024NDANDA216540Bridgebio Pharma

1. 1Diarrhoea4518%
2. 2Fatigue166.5%
3. 3Dizziness156.1%
4. 4Abdominal Discomfort145.7%
5. 5Blood Creatinine Increased124.9%
6. 6Nausea114.5%
7. 7Asthenia104.1%
8. 8Dyspnoea104.1%
9. 9Rash104.1%
10. 10Product Dose Omission Issue93.7%
11. 11Abdominal Pain Upper83.3%
12. 12Dysphagia83.3%
13. 13Weight Decreased83.3%
14. 14Gastrointestinal Disorder72.8%
15. 15Pruritus72.8%

The 10 most recently updated of 12 ClinicalTrials.gov registrations naming Acoramidis as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Phase 3 Study of ALXN2060 in Japanese Participants With Symptomatic ATTR-CM
  Completed · Phase 3 · Interventional · 25 enrolled · Alexion Pharmaceuticals, Inc.
  NCT04622046updated 2026-06-10
• A Study to Learn More About the Change in the Blood Levels of Transthyretin When Participants With Transthyretin Amyloidosis With Cardiomyopathy Switch From Tafamidis to Acoramidis
  Recruiting · Phase 4 · Interventional · 50 enrolled · Bayer
  NCT07298044updated 2026-06-08
• Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
  Recruiting · Phase 3 · Interventional · 587 enrolled · Eidos Therapeutics, a BridgeBio company
  NCT06563895updated 2026-05-12
• A Study to Learn About the Use of Acoramidis in Patients With a Heart Condition Called Transthyretin Amyloid Cardiomyopathy (ATTR-CM) in a Real-world Setting
  Recruiting · Observational · 2,000 enrolled · Bayer
  NCT07235462updated 2026-03-31
• Study to Assess the Effect of Food on a Single Dose of Acoramidis in Healthy Adult Participants
  Completed · Phase 1 · Interventional · 18 enrolled · Alexion Pharmaceuticals, Inc.
  NCT04958135updated 2026-03-06
• Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study
  Recruiting · Observational · 50 enrolled · University of Texas Southwestern Medical Center
  NCT07196839updated 2026-01-07
• Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers
  Recruiting · Phase 4 · Interventional · 200 enrolled · Alexion Pharmaceuticals, Inc.
  NCT07306949updated 2026-01-07
• Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants
  Active not recruiting · Phase 3 · Interventional · 389 enrolled · Eidos Therapeutics, a BridgeBio company
  NCT04988386updated 2025-10-14
• To Evaluate the Long-term Safety and Tolerability of Acoramidis in Participants With Newly Diagnosed ATTR-CM (ACT-EARLY OLE)
  Not yet recruiting · Phase 3 · Interventional · 250 enrolled · Eidos Therapeutics, a BridgeBio company
  NCT07116473updated 2025-08-11
• A Single Dose Study to Evaluate the Pharmacokinetics of Acoramidis Modified Release Formulations in Healthy Subjects
  Completed · Phase 1 · Interventional · 14 enrolled · Eidos Therapeutics, a BridgeBio company
  NCT04769479updated 2024-09-19

Frequently asked questions

How does Acoramidis work?

Acoramidis is a selective stabilizer of transthyretin (TTR). Acoramidis binds TTR at thyroxine binding sites and slows dissociation of the TTR tetramer into its constituent monomers, the rate-limiting step in amyloidogenesis.

What is Acoramidis used for?

According to FDA labeling, Acoramidis carries indications including: ATTRUBY is indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. ATTRUBY is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Acoramidis?

Acoramidis is classified as Other cardiac preparations, Transthyretin Stabilizer, Cytochrome P450 2C9 Inhibitors, Transthyretin Stabilizers.

What are the brand names for Acoramidis?

Acoramidis is marketed under brand names including Attruby.

What are the contraindications for Acoramidis?

Acoramidis labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.