Acoramidis Demonstrates Real-World Benefit Versus Tafamidis in ATTR-CM, Reducing Risk of Clinical Worsening
New real-world comparative data suggest acoramidis may offer earlier and greater clinical stabilization than tafamidis in ATTR-CM, though follow-up is short.
Rhea-AI Summary
BridgeBio Pharma (BBIO) reported peer-reviewed real-world data showing acoramidis reduced early clinical worsening versus tafamidis in adults with ATTR-CM.
In a retrospective U.S. claims study of newly treated patients, acoramidis was associated with a 34% lower risk of a composite endpoint (diuretic intensification, heart failure hospitalization, all-cause mortality; HR 0.66; p=0.046) compared with tafamidis over a mean 4.6‑month follow-up. Acoramidis was also linked to a 43% reduction in risk of diuretic intensification (HR 0.57; p=0.021) and a 48% reduction in initiation or dose-equivalent escalation of oral loop diuretics (HR 0.52; p=0.014). Separation in outcomes appeared within weeks of treatment initiation and widened over time. Background heart failure therapies were well balanced between groups, and multiple falsification analyses were nonsignificant. The company highlighted real-world evidence limitations and noted longer-term data and an additional independent EHR-based study are forthcoming.
Positive
- 34% reduction in composite clinical worsening risk vs tafamidis (HR 0.66; p=0.046) over 4.6 months
- 43% lower risk of diuretic intensification with acoramidis vs tafamidis (HR 0.57; p=0.021)
- 48% reduction in initiation or escalation of oral loop diuretics vs tafamidis (HR 0.52; p=0.014)
- Early and increasing separation of Kaplan-Meier curves suggests rapid and durable treatment effect within study window
- Fewer acoramidis-treated patients started alternative ATTR-CM therapy (4.6% vs 7.7% with tafamidis)
- Multiple falsification analyses were nonsignificant, supporting robustness of comparative findings within study design
Negative
- Mean follow-up was only 4.6 months, limiting insight into longer-term outcomes and durability
- Real-world claims design has inherent limitations, including limited capture of cardiac biomarkers and disease severity measures
- Attruby (acoramidis) showed higher rates of diarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) vs placebo in prior data
Key Figures
- Composite clinical worsening risk reduction
- 34% (HR 0.66; p=0.046)
- Acoramidis versus tafamidis
- Diuretic intensification risk reduction
- 43% (HR 0.57; p=0.021)
- Acoramidis versus tafamidis
- Oral loop diuretic initiation or escalation reduction
- 48% (HR=0.52; p=0.014)
- Acoramidis versus tafamidis
- Acoramidis study participants
- 170 individuals
- Weighted analysis
- Tafamidis study participants
- 448 individuals
- Weighted analysis
- Mean follow-up
- 4.6 months
- Study analysis
Key Terms
ttr stabilizers medical
kaplan-meier technical
sglt2i medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
- In the first peer-reviewed, real-world, contemporary, comparative effectiveness study of TTR stabilizers in ATTR-CM using U.S. claims data, acoramidis was associated with a statistically and clinically significant
- Acoramidis was also associated with a significant
- Separation in clinical outcomes emerged within weeks of treatment initiation and increased over time, reinforcing a consistent early treatment effect and durable clinical stabilization
- Findings underscore acoramidis’s differentiated profile, with potential to inform frontline treatment decisions and support switching individuals to acoramidis
- An additional, independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data is expected to be released at an upcoming 2026 medical meeting
PALO ALTO, Calif., Sept. 23, 2026 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage, multi-product biopharmaceutical company focused on developing medicines for genetic conditions, today announced results published in Cardiology and Therapy from the first peer-reviewed, contemporary real-world comparative effectiveness retrospective study of transthyretin (TTR) stabilizers in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM), demonstrating that acoramidis was associated with significantly lower risk of early clinical worsening compared to tafamidis. This represents the first peer-reviewed, real-world evidence differentiating clinical outcomes between approved TTR stabilizers, reinforcing acoramidis’s differentiated clinical profile in present-day practice.
“These findings further position acoramidis as a differentiated TTR stabilizer in contemporary clinical practice,” said Richard Wright, M.D., M.A.C.C. of the Pacific Heart Institute, U.S. “The significant reduction in diuretic intensification, an early indicator of worsening heart failure, points to improved disease control and clinical stability. The progressive nature of ATTR-CM is evident in all Phase 3 trials and is seen again here. This demonstrates the need for clinicians to proactively mitigate disease progression, and these data have the potential to meaningfully inform treatment selection in newly diagnosed patients and in patients currently on other treatments.”
In this analysis of newly treated individuals living with ATTR-CM, acoramidis showed early, consistent, and clinically meaningful advantages compared to tafamidis across key measures of disease progression during a mean follow-up of 4.6 months.
The key findings from the study comparing acoramidis benefit versus tafamidis in individuals with ATTR-CM:
34% statistically and clinically significant reduction in risk of composite clinical worsening (HR 0.66; p=0.046), including diuretic intensification, heart failure hospitalization, and all-cause mortality43% reduction in risk of diuretic intensification (HR 0.57; p=0.021), an established early marker of worsening heart failure and predictor of hospitalization and mortality; diuretic intensification was rigorously defined as initiation or dose-equivalent escalation of oral loop diuretics, parenteral loop diuretics use, or addition of a thiazide-type diuretic48% reduction in initiation or dose-equivalent escalation of oral loop diuretics (HR=0.52; p=0.014)- Rapid benefit, with separation of Kaplan-Meier curves observed within weeks of treatment initiation and increasing over time
- Fewer individuals with acoramidis initiated alternative ATTR-CM therapy as compared to those treated with tafamidis (
4.6% vs7.7% , respectively), consistent with findings of improved clinical stability with acoramidis - Background heart failure therapies in this study were well balanced across arms and reflect contemporary practice: 43
-44% SGLT2i, 36-39% MRA - Multiple falsification analyses testing for residual bias were nonsignificant
The study leveraged a retrospective, longitudinal, new-user, active-comparator design using U.S. claims data and incorporated a unique dataset linking Komodo Healthcare Map U.S. claims with Claritas hub and specialty pharmacy data. After weighting, the analysis included 170 individuals with acoramidis and 448 individuals with tafamidis newly initiating treatment between Dec 2024-April 2025 and followed through July 2025, with a mean follow-up of approximately 4.6 months.
Acoramidis is approved as Attruby® by the U.S. FDA and is approved as BEYONTTRA® by the European Medicines Agency (EMA), Japanese Pharmaceuticals and Medical Devices Agency, Swissmedic, the Swiss Agency for Therapeutic Products, the UK Medicines and Healthcare Products Regulatory Agency, and the Brazilian Health Regulatory Agency (ANVISA) with all labels specifying near-complete stabilization of TTR.
Additional data on the real-world benefit of acoramidis versus tafamidis in individuals living with ATTR-CM is planned for fall medical meetings and scientific publications, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, expected to be released at an upcoming 2026 medical meeting.
As with all real-world evidence studies, these findings should be interpreted in the context of inherent limitations of administrative claims data, including limited capture of cardiac biomarkers and other clinical measures of disease severity. Follow-up was limited given the recent approval of acoramidis, and longer-term data are needed to further add to these findings.
About Attruby® (acoramidis)
INDICATION
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.
IMPORTANT SAFETY INFORMATION
Adverse Reactions
Diarrhea (
About BridgeBio
BridgeBio exists to develop transformative medicines for genetic conditions. Millions of people worldwide living with genetic conditions lack treatment options, often because drug development for small patient populations can be commercially challenging. We aim to bridge the gap between advancements in genetic science and meaningful medicines for underserved patient populations. Our decentralized, hub-and-spoke model is designed for speed, precision, and scalability. Autonomous and empowered teams focus on individual conditions, while a central hub provides the clinical, regulatory, and commercial capabilities needed to bring innovation to market. For more information, visit bridgebio.com and follow us on LinkedIn, X, Facebook, Instagram, YouTube, and TikTok.
BridgeBio Forward-Looking Statements
This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. BridgeBio intends these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements include statements regarding the potential clinical significance and therapeutic implications of the real-world data regarding acoramidis, including the potential for the findings to inform frontline treatment decisions and treatment selection for newly diagnosed patients and patients currently receiving other treatments, and to support switching individuals to acoramidis; and BridgeBio’s plans and expectations regarding the presentation and publication of additional real-world data regarding acoramidis, including an independent real-world study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data expected to be released at an upcoming 2026 medical meeting. Although the Company believes that its plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, the Company can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, the risk that results from retrospective, observational, real-world evidence studies, including studies based on administrative claims data, may be subject to confounding, selection bias, residual bias, incomplete or inaccurate data, limited follow-up or other limitations and may not be predictive of future clinical outcomes or treatment effects; that the observed associations and differences between acoramidis and tafamidis may not be replicated in additional analyses, independent studies or longer-term data or may not translate into improved long-term clinical outcomes; that the findings may not meaningfully inform treatment selection or support switching patients to acoramidis; that additional real-world studies, including the independent study evaluating the effectiveness of acoramidis versus tafamidis using electronic health records data, may yield results that differ from or do not confirm the findings described in this press release; that plans or timing for future medical meeting presentations or scientific publications may change; the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Middle East, increasing rates of inflation and changing interest rates, on business operations and expectations, as well as those risks set forth in the Risk Factors section of the Company’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K and the Company’s other filings with the U.S. Securities and Exchange Commission. Moreover, the Company operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of the Company’s management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, BridgeBio assumes no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
BridgeBio Media Contact:
Kaitlyn Reilly, Director, Communications
contact@bridgebio.com
(650) 789-8220
BridgeBio Investor Contact:
Kristen Kelleher, Director, Investor Relations
ir@bridgebio.com
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What study design was used to compare acoramidis and tafamidis in ATTR-CM?
The analysis used a retrospective, longitudinal, new-user, active-comparator design based on U.S. claims data. It linked the Komodo Healthcare Map claims dataset with Claritas hub and specialty pharmacy data to identify adults with ATTR-CM newly initiating acoramidis or tafamidis and followed them for clinical worsening outcomes.
How many patients were included in the acoramidis versus tafamidis comparison and over what period?
After weighting, the study included 170 individuals treated with acoramidis and 448 individuals treated with tafamidis. Patients newly initiated therapy between December 2024 and April 2025 and were followed through July 2025, with a mean follow-up duration of approximately 4.6 months.
Were background heart failure therapies balanced between treatment groups?
Background heart failure therapies were described as well balanced across the acoramidis and tafamidis arms and reflective of contemporary practice, with 43–44% of patients on SGLT2 inhibitors and 36–39% on mineralocorticoid receptor antagonists.
What additional real-world data on acoramidis are expected?
The company plans further presentations and publications on the real-world benefit of acoramidis versus tafamidis, including an independent study using electronic health records to evaluate effectiveness. This EHR-based analysis is expected to be released at an upcoming 2026 medical meeting.
For what indication is Attruby (acoramidis) currently approved?
Attruby, the U.S. brand name for acoramidis, is indicated as a transthyretin stabilizer for treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults, to reduce cardiovascular death and cardiovascular-related hospitalization.
What adverse reactions have been observed with Attruby in clinical use?
In prior data comparing Attruby to placebo, diarrhea occurred in 11.6% of patients on Attruby versus 7.6% on placebo, and upper abdominal pain occurred in 5.5% versus 1.4%, respectively. Most events were mild and resolved without treatment discontinuation, and discontinuation rates due to adverse events were similar (9.3% for Attruby vs 8.5% for placebo).