Latest news with #Acoramidis
Yahoo
20-05-2025
- Health
- Yahoo
Acoramidis Reduced Incidence of Atrial Fibrillation Events in Patients with ATTR-CM
- In a post-hoc analysis of ATTRibute-CM, acoramidis reduced the annual frequency of CVH due to AF/AFL by 43% compared to placebo and reduced the incidence of new-onset AF/AFL by 17% in the subgroup with no prior history of AF compared to placebo - In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRwt-CM patients: - In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 - Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA® by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency and UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., May 20, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, presented data from ATTRibute-CM, highlighting the reduced incidence of atrial fibrillation (AF) events in the overall ATTR-CM population. These data were presented in a moderated ePoster at the Annual Congress of the Heart Failure Association of the ESC (Heart Failure 2025), taking place in Belgrade, Serbia from May 17 - 20, 2025. Acoramidis is a selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. 'The reduction in new-onset atrial fibrillation and AF-related hospitalizations represents an important finding for the ATTR-CM community. I am encouraged by the growing body of data from the ATTRibute-CM study, which adds to the understanding of acoramidis and its potential impact on clinical outcomes for patients. Furthermore, the observed reductions in hospitalizations and mortality, along with improvements in functional capacity and quality of life, suggest that acoramidis may offer benefit to both variant and wild-type ATTR-CM patients who have limited treatment options,' said Kevin Alexander, M.D. of Stanford University School of Medicine, USA. 'These data support further consideration of acoramidis as a promising front-line therapy for ATTR-CM, particularly for patients with the hereditary form of the disease, who often face rapid and severe progression.' Details from the post-hoc analysis on incidence of AF in ATTRibute-CM included: Acoramidis Treatment Is Associated with a Lower Incidence of Atrial Fibrillation-related Events in Patients with ATTR-CM: A Post-hoc Analysis of the ATTRibute-CM Trial, presented by Dr. Alexander AF is a common complication of ATTR-CM, observed in up to 70% of patients, and the onset of AF is associated with an increased risk of cardiovascular-related hospitalizations (CVH) In ATTRibute-CM, a 43% relative risk reduction in the annual frequency of CVH due to AF/atrial flutter (AFL) was observed with acoramidis relative to placebo. In the subgroup who had no prior history of AF, a 17% lower incidence of new-onset AF/AFL was reported with acoramidis compared to placebo These findings show the potential of acoramidis to reduce both disease progression, as indicated by a lower incidence of new-onset AF/AFL, and CVH morbidity caused by AF/AFL, in patients with ATTR-CM In addition to the moderated ePoster, three analyses were shared on the strong clinical outcomes in ATTRv-CM versus placebo. ATTRv-CM is associated with early age of disease onset with more advanced heart failure symptoms, which often leads to a poorer prognosis than those with wild-type ATTR-CM (ATTRwt-CM). These findings included: Acoramidis Improves Serum TTR Levels in Patients with Wild-type or Variant Transthyretin Amyloid Cardiomyopathy: Results from ATTRibute-CM, presented by Anique Ducharme, M.D. of Université de Montréal, CAN In both subgroups of ATTRv-CM and ATTRwt-CM, acoramidis treatment induced a rapid increase in serum TTR levels, a measure of TTR stability, by Day 28, with comparable serum TTR levels achieved in both subgroups from Day 28 through Month 30. Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature1 Effect of Acoramidis on Functional Capacity and Quality of Life in Patients with Variant ATTR-CM: Results from ATTRibute-CM, presented by Marianna Fontana, M.D. of University College London, UK Data from ATTRibute-CM showed that when acoramidis was administered for 30 months, participants with ATTRv-CM had a clinically significant slower decline in functional capacity and quality of life compared with placebo, consistent with the overall results in both ATTRv-CM and ATTRwt-CM At Month 30, the mean difference between acoramidis and placebo treatment groups in the change from baseline in 6-minute walk distance was 86.7 meters (p = 0.0048) in favor of acoramidis and in the change from baseline in KCCQ-OS at Month 30, was 20.3 points (p = 0.0019) in favor of acoramidis, in patients with ATTRv-CM Effect of Acoramidis on All-cause Mortality, Cardiovascular Hospitalization and NT-proBNP in Variant ATTR-CM: Results from ATTRibute-CM, presented by Marianna Fontana, M.D. of University College London, UK In ATTRibute-CM, acoramidis treatment administered for 30 months led to a substantial reduction (>50%) in the composite of all-cause mortality (ACM)/CVH, ACM and CVH in participants with ATTRv-CM compared to placebo. This improvement was accompanied by favorable effects on N-terminal pro-B-type natriuretic peptide (NT-proBNP) levels 'Based on the findings from ATTRibute-CM, we believe that acoramidis has the potential to enable patients with both variant and wild-type ATTR-CM to live longer, healthier lives, especially for those with variant ATTR-CM who typically have a poorer prognosis. We observed both a striking reduction in the frequency of cardiovascular hospitalizations (which included clinic or emergency department visits for urgent heart failure management) and a clinically important and statistically significant reduction in all-cause mortality in the important subgroup. These clinical outcomes were further mirrored in robust improvements in functional capacity, quality of life, and biomarkers of heart failure severity,' said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. 'Given these compelling results, acoramidis should be considered as first-line treatment for newly diagnosed patients, and those currently on other therapies could be switched to acoramidis to maximize their potential to achieve such benefits.' Additional acoramidis moderated ePosters at Heart Failure 2025 included: Disease Progression Among Patients Receiving Tafamidis for ATTR-CM in a Real-world Setting, presented by Daniel P. Judge, M.D. of Medical University of South Carolina, USA This analysis suggests disease progression despite treatment with tafamidis in ATTR-CM. CVH was frequent, with approximately 1 in 5 tafamidis-treated patients hospitalized in the first six months of therapy. As more therapeutic options become available, measuring the clinical effectiveness of therapies in a real-world setting will be important to help inform physicians and patients when making treatment decisions Cause of Death in Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM): Findings from the ATTRibute-CM Study, presented by Laura Obici, M.D. of University of Pavia, ESP In the ATTRibute-CM study, total deaths were numerically lower with acoramidis compared with placebo. The relative risk reduction of 30% in cardiovascular-related mortality by Month 30 was driven predominantly by a reduction in heart failure-related deaths Time from First Recorded Clinical Manifestation to Diagnosis of Transthyretin Amyloid Cardiomyopathy: A Retrospective Cohort Study Using U.S. Claims Data, presented by Joshua Mitchell, M.D., Washington University School of Medicine in St. Louis, USA The median time from the first documented clinical manifestation to ATTR-CM diagnosis was almost 5 years, and over 2 years from the first heart failure diagnosis. This demonstrates that the patient journey to an ATTR-CM diagnosis can be prolonged and challenging, which potentially leads to more severe disease at diagnosis. Understanding the factors contributing to diagnostic delays is important to improving diagnostic pathways and patient outcomes Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR. More data on the benefit of Attruby for ATTR-CM patients is planned for future medical meetings. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. 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 (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBioBridgeBio Pharma (BridgeBio; NASDAQ:BBIO) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn, Twitter, Facebook, and YouTube. 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,' 'could,' 'estimates,' 'expects,' 'hopes,' 'intends,' 'may,' 'plans,' 'projects,' 'potential,' '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, including statements regarding the potential impact of acoramidis on clinical outcomes for patients, the possibility that acoramidis may offer benefit to both variant and wild-type ATTR-CM patients, the consideration of acoramidis as a promising front-line therapy for ATTR-CM, the potential of acoramidis to reduce disease progression and cardiovascular hospitalization morbidity caused by AF/AFL, BridgeBio's belief that acoramidis could help patients live longer, healthier lives—especially those with variant ATTR-CM—and the view that acoramidis should be considered as a first-line treatment or as a replacement for current therapies to maximize patient benefit, reflect BridgeBio's current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions it has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies as reflected in or suggested by these forward-looking statements are reasonable, it can give no assurance that such 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 risks associated with BridgeBio's dependence on third parties for development; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet regulatory requirements or are sufficient for continued development, review, or approval; and whether other regulatory agencies agree with BridgeBio's strategies or data interpretations. These risks also include impacts from global health emergencies, such as delays in regulatory reviews and other activities, manufacturing and supply chain interruptions, adverse effects on healthcare systems, and disruption of the global economy; and the impacts of macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing inflation rates, and fluctuating interest rates on BridgeBio's operations and expectations. Additional risks are described in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio 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 BridgeBio'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 these statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@ (650)-789-8220 BridgeBio Investor Contact:Chinmay Shukla, VP Strategic Financeir@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
19-05-2025
- Health
- Yahoo
Early and Sustained Increase in Serum TTR Levels by Acoramidis Independently Predicted Improved Survival in the ATTRibute-CM Study
- For each 5-mg/dL increase in serum TTR level within 28 days of starting treatment, the relative risk reduction of mortality was up to 31.6% through Month 30, confirming the hypothesis that ever better levels of stabilization achieved by treatment with acoramidis, a near-complete (≥90%) TTR stabilizer, lead to ever better clinical outcomes - ATTRibute-CM is the only study to demonstrate a direct association between a prompt, sustained increase in serum TTR and survival in patients with ATTR-CM - The open-label extension data for all ATTRibute-CM participants at Month 42 showed that rapid, sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in ACM and CVH (including urgent outpatient treatment for heart failure exacerbations) - In the ATTRibute-CM study, acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date in both ATTRv-CM and ATTRwt-CM patients: - In as few as 3 months, the time to first event (ACM or CVH) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 - Acoramidis is approved as Attruby™ by the U.S. FDA and is approved as BEYONTTRA® by the European Commission, the Japanese Pharmaceuticals and Medical Devices Agency, and the UK Medicines and Healthcare Products Regulatory Agency PALO ALTO, Calif., May 19, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, published data showing that an early, sustained increase in serum transthyretin (TTR) levels predicted improved survival in ATTRibute-CM, its Phase 3 trial of acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM). These findings were published in Journal of the American College of Cardiology (JACC) in the Special Focus Issue: Amyloid. Acoramidis is a selective, small molecule, orally administered, near-complete (≥90%) TTR stabilizer. These findings further support the thesis that ever better increases in serum TTR lead to ever better clinical outcomes and that early elevations in serum TTR are an important prognostic marker to inform treatment selection. 'Patients with ATTR-CM have progressive amyloid accumulation in the heart, which when untreated manifests as progressive heart failure, arrhythmias, and eventually can result in death. Increases in serum TTR seen with acoramidis therapy within 28 days of initiation and that were sustained with therapy, were associated with a decrease in all-cause mortality independent of baseline risk among subjects in the ATTRibute-CM trial. The increase in serum TTR is hypothesized to be due to a leftward shift in amyloidogenic TTR to a more stable tetrameric TTR. This is the first concrete evidence that there is a link between this rapid increase in serum TTR and survival. Such data may inform clinical practice, as early and sustained increases in serum TTR could represent a new potential ATTR disease-specific and prognostic biomarker that may further inform clinical decisions in optimizing care for ATTR-CM patients,' said Mathew Maurer, M.D. of Columbia University Irving Medical Center. In patients with ATTR-CM, aging or an inherited variant can cause tetrameric TTR to destabilize and misfold, causing buildup of amyloid fibrils in the organs, specifically in the heart. These data demonstrate that by using acoramidis, a selective near-complete TTR stabilizer to bind serum TTR, a rapid and sustained increase in tetrameric TTR was independently associated with an improvement in overall survival, even after adjustment for known predictors like TTR variant status, baseline New York Heart Association (NYHA) functional class, baseline National Amyloidosis Centre (NAC) stage, and baseline serum TTR levels. Findings from the analysis included: Treatment with acoramidis resulted in a sharp, significant early rise in serum TTR levels (mean 9.1 mg/dL) within 28 days which was sustained throughout the 30-month treatment period For every 5-mg/dL increase in serum TTR level, the Cox proportional hazards model predicted a relative risk reduction of mortality of 26.6% and the logistic model predicted a relative reduction of 31.6% in odds of death through Month 30 An early increase in serum TTR levels on Day 28 of dosing was associated with reduced all-cause mortality (ACM) in univariate analysis, an association which persisted in multivariate analysis independent of TTR variant status, baseline NYHA functional class, baseline NAC stage, and baseline serum TTR levels Logistic modeling demonstrated that among participants treated with acoramidis, the early increase in serum TTR was associated with reduced ACM, whereas there was no prompt and sustained increase in serum TTR observed in participants treated with placebo Causal mediation analysis showed evidence that the acoramidis treatment effect on ACM probability through month 30 was fully mediated by the observed prompt and sustained increase in serum TTR 'This landmark analysis adds an incredibly important proof point to acoramidis's repository of compelling data that higher serum TTR levels are directly correlated with and mediate a reduction in mortality risk.' said Jonathan Fox, M.D., Ph.D., Chief Medical Officer of BridgeBio Cardiorenal. 'We believe that this will be an important measure for physicians treating ATTR-CM to consider and will be encouraging information for new and existing patients when reviewing options to treat their condition.' Data from 42 months sustained treatment in the open-label extension study from ATTRibute-CM showed that rapid and sustained TTR stabilization from acoramidis demonstrated statistically significant reductions in both ACM and cardiovascular-related hospitalizations (CVH, which included urgent outpatient treatment for heart failure exacerbations). The continued curve separation of the composite endpoint of ACM and CVH emphasizes the importance of early and continuous treatment resulting in early and sustained clinical benefits. These data further underscore the hypothesis that ever better levels of stabilization lead to ever better clinical outcomes and emphasizes the importance of a prognostic biomarker, serum TTR, to inform decision making for patient care. Acoramidis is approved as Attruby by the U.S. FDA and is approved as BEYONTTRA by the European Commission, Japanese Pharmaceuticals and Medical Devices Agency, and UK Medicines and Healthcare Products Regulatory Agency with all labels specifying near-complete stabilization of TTR. 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 (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). About BridgeBio Pharma, Pharma, Inc. (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn, Twitter, Facebook, and YouTube. BridgeBio Forward-Looking StatementsThis 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,' 'could,' 'estimates,' 'expects,' 'hopes,' 'intends,' 'may,' 'plans,' 'projects,' 'potential,' '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, including statements regarding the potential for sustained increases in serum TTR to serve as a disease-specific and prognostic biomarker in ATTR-CM, the belief that early elevations in serum TTR are an important prognostic marker to inform treatment selection, the hypothesis that improved serum TTR stabilization is correlated with better clinical outcomes, and expectations about how these findings may guide physician decision-making and patient care, reflect BridgeBio's current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions it has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies as reflected in or suggested by these forward-looking statements are reasonable, it can give no assurance that such 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 risks associated with BridgeBio's dependence on third parties for development; regulatory authorities requiring additional studies or data to support the continued or expanded commercialization of acoramidis; whether data and results meet regulatory requirements or are sufficient for continued development, review, or approval; and whether other regulatory agencies agree with BridgeBio's strategies or data interpretations. These risks also include impacts from global health emergencies, such as delays in regulatory reviews and other activities, manufacturing and supply chain interruptions, adverse effects on healthcare systems, and disruption of the global economy; and the impacts of macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing inflation rates, and fluctuating interest rates on BridgeBio's operations and expectations. Additional risks are described in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q, and other filings with the U.S. Securities and Exchange Commission. Moreover, BridgeBio 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 BridgeBio'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 these statements. Except as required by applicable law, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, Executive Vice Presidentcontact@ BridgeBio Investor Contact:Chinmay Shukla, VP Strategic Financeir@
Yahoo
29-04-2025
- Business
- Yahoo
UK MHRA authorises BridgeBio's acoramidis for ATTR-CM
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for BridgeBio Pharma's acoramidis under the name Beyonttra, for treating wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is an orally administered selective small molecule that stabilises transthyretin (TTR) by more than 90%. This authorisation was supported by the outcomes from the pivotal ATTRibute-CM Phase III trial, which demonstrated cardiovascular benefits. In the trial, the therapy's safety and efficacy were assessed in 632 symptomatic ATTR-CM subjects, and they were given either the therapy or a placebo for a 30-month period. The primary clinical endpoints were met at month 30, showing a decrease in cardiovascular-related hospitalisations, preservation of functional capacity, improved survival and quality of life for subjects. The therapy received the US Food and Drug Administration's (FDA) approval under the name Attruby in November 2024, and as Beyonttra by the European Commission (EC) in February 2025. It was also approved in Japan by the Ministry of Health, Labour, and Welfare in March 2025. In the UK, Bayer will manage all commercial activities for the therapy. In March 2024, the company and Bayer entered a partnership granting the latter exclusive rights to commercialise the therapy in Europe. According to the licencing agreement, BridgeBio will obtain tiered royalties starting in the low-thirties percent on UK sales after commercialisation efforts commence. ATTR-CM is a fatal condition that causes heart failure due to restrictive cardiomyopathy. BridgeBio Cardiorenal chief medical officer and president Jonathan Fox stated: 'We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to Beyonttra since they are in great need of new disease-modifying treatments for their condition. 'We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world." "UK MHRA authorises BridgeBio's acoramidis for ATTR-CM" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
28-04-2025
- Business
- Yahoo
BEYONTTRA® (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved by the UK Medicines and Healthcare Products Regulatory Agency to Treat ATTR-CM
- The UK approval is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date - In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH)) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 - Acoramidis is the first and only approved ATTR-CM treatment in the U.S., EU, UK and Japan that all have a label specifying near-complete stabilization (≥90%) - Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature1 - BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of Beyonttra in the UK PALO ALTO, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, today announced the Medicines and Healthcare products Regulatory Agency has granted marketing authorization in the United Kingdom (UK) for acoramidis, under the brand name BEYONTTRA®, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is a selective small molecule, orally administered near-complete (≥90%) transthyretin (TTR) stabilizer. ATTR-CM is a progressive fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure. Bayer will be responsible for all commercial activity for acoramidis in the UK. 'ATTR-CM is a progressive and debilitating disease that poses significant challenges not only for patients but also for the healthcare systems. The condition profoundly impacts patients' quality of life. Symptoms attributable to amyloidosis are usually nonspecific, varied and associated with low awareness, frequently resulting in delayed or completely missed diagnosis,2 which may lead to delayed treatment and a worse prognosis. In the absence of intervention, ATTR-CM causes progressive heart failure leading to increased hospitalizations and escalating healthcare costs and is ultimately fatal,3-5' said Julian Gillmore, M.D., Ph.D., University College London's Centre for Amyloidosis, UK. 'The UK authorization of Beyonttra is welcome news for eligible patients living with the condition. Physicians in the UK now have another treatment option to slow the progression of symptoms and improve outcomes for patients with ATTR-CM.' The approval in the UK is based on results of the pivotal ATTRibute-CM Phase 3 study of acoramidis, which showed clear benefits on cardiovascular outcomes. ATTRibute-CM evaluated the efficacy and safety of acoramidis in 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR who were randomized 2:1 to receive acoramidis or placebo for 30 months. The study met its primary clinical endpoints at month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients. 'We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to BEYONTTRA since they are in great need of new disease-modifying treatments for their condition,' said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. 'We appreciate the time and commitment of every clinical trial participant and their families, and the dedicated support of the physicians and scientists involved in the clinical program. This important milestone would not have been possible without their commitment to the program. We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world." Acoramidis was approved as Attruby™ by the U.S. FDA in November 2024 and was approved as BEYONTTRA by the European Commission in February 2025 and the Japanese Ministry of Health, Labour, and Welfare (MHLW) Agency in March 2025 with all labels specifying near-complete stabilization of TTR. In March 2024, BridgeBio and Bayer initiated a collaboration for acoramidis, which granted Bayer exclusive commercialization rights in Europe. Based on terms of the licensing agreement, BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in the UK following initiation of commercialization efforts. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102.2Rintell et al. Orphanet J Rare Dis. (2021) 16:70. 3Rozenbaum MH, et al. Impact of delayed diagnosis and misdiagnosis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM): a targeted literature review. Cardiology and therapy. 2021;10:141-59.4Mallus MT and Rizzello V. Treatment of amyloidosis: present and future. 2023;21;25(Suppl B):B99-B103.5Jain A, Zahra F. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Updated 27 April 2023. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: Last accessed: March 2025. About BEYONTTRABEYONTTRA is an orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) indicated for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). For full prescribing information, please refer to the Summary of Product Characteristics (SmPC) on the Medicines and Healthcare products Regulatory Agency website at About Attruby™ (acoramidis)INDICATIONAttruby 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 INFORMATIONAdverse ReactionsDiarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). ▼: This medicine is subject to additional monitoring. This will allow quick identification of new safety information. About BridgeBio BridgeBio is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn and Twitter. 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, including statements relating to the impact of Beyonttra on clinical outcomes; and the potential benefits of Beyonttra, including its ability to reduce cardiovascular-related hospitalization, improve survival, and preserve functional capacity and quality of life, reflect BridgeBio's current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies, as reflected in or suggested by these forward-looking statements, are reasonable, BridgeBio 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 risks associated with BridgeBio's dependence on third parties for development, manufacture, and commercialization activities related to Beyonttra; government and third-party payor actions; risks and uncertainties relating to competitive products and other changes that may limit demand for Beyonttra; the risk that regulatory authorities may require additional studies or data to support the continued commercialization of Beyonttra; the risk that drug-related adverse events may be observed during commercialization or clinical development; the risk that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review, or approval; the risk of other regulatory agencies not agreeing with BridgeBio's regulatory approval strategies, components of BridgeBio's filings (such as clinical trial designs, conduct, and methodologies), or the sufficiency of data submitted; the continuing success of its collaborations, including compliance with applicable regulations for the purchase, distribution, storage, export, and sale of active pharmaceutical ingredients and medicinal products; uncertainty regarding any impacts due to global health emergencies, including delays in regulatory review, manufacturing, and supply chain interruptions; adverse effects on healthcare systems and disruption of the global economy; the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip; and increasing rates of inflation and changing interest rates on BridgeBio's business operations and expectations. These risks, as well as those set forth in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K and its other filings with the U.S. Securities and Exchange Commission, should be carefully considered. Moreover, BridgeBio operates in a highly 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 BridgeBio'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 publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@ Chinmay Shukla, VP Strategic Financeir@
Associated Press
28-04-2025
- Business
- Associated Press
BEYONTTRA® (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved by the UK Medicines and Healthcare Products Regulatory Agency to Treat ATTR-CM
- The UK approval is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date - Acoramidis is the first and only approved ATTR-CM treatment in the U.S., EU, UK and Japan that all have a label specifying near-complete stabilization (≥90%) - Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature1 - BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of Beyonttra in the UK PALO ALTO, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ('BridgeBio' or the 'Company'), a new type of biopharmaceutical company focused on genetic diseases, today announced the Medicines and Healthcare products Regulatory Agency has granted marketing authorization in the United Kingdom (UK) for acoramidis, under the brand name BEYONTTRA®, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is a selective small molecule, orally administered near-complete (≥90%) transthyretin (TTR) stabilizer. ATTR-CM is a progressive fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure. Bayer will be responsible for all commercial activity for acoramidis in the UK. 'ATTR-CM is a progressive and debilitating disease that poses significant challenges not only for patients but also for the healthcare systems. The condition profoundly impacts patients' quality of life. Symptoms attributable to amyloidosis are usually nonspecific, varied and associated with low awareness, frequently resulting in delayed or completely missed diagnosis,2 which may lead to delayed treatment and a worse prognosis. In the absence of intervention, ATTR-CM causes progressive heart failure leading to increased hospitalizations and escalating healthcare costs and is ultimately fatal,3-5' said Julian Gillmore, M.D., Ph.D., University College London's Centre for Amyloidosis, UK. 'The UK authorization of Beyonttra is welcome news for eligible patients living with the condition. Physicians in the UK now have another treatment option to slow the progression of symptoms and improve outcomes for patients with ATTR-CM.' The approval in the UK is based on results of the pivotal ATTRibute-CM Phase 3 study of acoramidis, which showed clear benefits on cardiovascular outcomes. ATTRibute-CM evaluated the efficacy and safety of acoramidis in 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR who were randomized 2:1 to receive acoramidis or placebo for 30 months. The study met its primary clinical endpoints at month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients. 'We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to BEYONTTRA since they are in great need of new disease-modifying treatments for their condition,' said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. 'We appreciate the time and commitment of every clinical trial participant and their families, and the dedicated support of the physicians and scientists involved in the clinical program. This important milestone would not have been possible without their commitment to the program. We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world.' Acoramidis was approved as Attruby™ by the U.S. FDA in November 2024 and was approved as BEYONTTRA by the European Commission in February 2025 and the Japanese Ministry of Health, Labour, and Welfare (MHLW) Agency in March 2025 with all labels specifying near-complete stabilization of TTR. In March 2024, BridgeBio and Bayer initiated a collaboration for acoramidis, which granted Bayer exclusive commercialization rights in Europe. Based on terms of the licensing agreement, BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in the UK following initiation of commercialization efforts. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. 2Rintell et al. Orphanet J Rare Dis. (2021) 16:70. 3Rozenbaum MH, et al. Impact of delayed diagnosis and misdiagnosis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM): a targeted literature review. Cardiology and therapy. 2021;10:141-59. 4Mallus MT and Rizzello V. Treatment of amyloidosis: present and future. 2023;21;25(Suppl B):B99-B103. 5Jain A, Zahra F. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Updated 27 April 2023. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: Last accessed: March 2025. About BEYONTTRA BEYONTTRA is an orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) indicated for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). For full prescribing information, please refer to the Summary of Product Characteristics (SmPC) on the Medicines and Healthcare products Regulatory Agency website at 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 (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). ▼: This medicine is subject to additional monitoring. This will allow quick identification of new safety information. About BridgeBio BridgeBio is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio's pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit and follow us on LinkedIn and Twitter. 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, including statements relating to the impact of Beyonttra on clinical outcomes; and the potential benefits of Beyonttra, including its ability to reduce cardiovascular-related hospitalization, improve survival, and preserve functional capacity and quality of life, reflect BridgeBio's current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies, as reflected in or suggested by these forward-looking statements, are reasonable, BridgeBio 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 risks associated with BridgeBio's dependence on third parties for development, manufacture, and commercialization activities related to Beyonttra; government and third-party payor actions; risks and uncertainties relating to competitive products and other changes that may limit demand for Beyonttra; the risk that regulatory authorities may require additional studies or data to support the continued commercialization of Beyonttra; the risk that drug-related adverse events may be observed during commercialization or clinical development; the risk that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review, or approval; the risk of other regulatory agencies not agreeing with BridgeBio's regulatory approval strategies, components of BridgeBio's filings (such as clinical trial designs, conduct, and methodologies), or the sufficiency of data submitted; the continuing success of its collaborations, including compliance with applicable regulations for the purchase, distribution, storage, export, and sale of active pharmaceutical ingredients and medicinal products; uncertainty regarding any impacts due to global health emergencies, including delays in regulatory review, manufacturing, and supply chain interruptions; adverse effects on healthcare systems and disruption of the global economy; the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip; and increasing rates of inflation and changing interest rates on BridgeBio's business operations and expectations. These risks, as well as those set forth in the Risk Factors section of BridgeBio's most recent Annual Report on Form 10-K and its other filings with the U.S. Securities and Exchange Commission, should be carefully considered. Moreover, BridgeBio operates in a highly 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 BridgeBio'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 publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact: Bubba Murarka, EVP Communications Chinmay Shukla, VP Strategic Finance [email protected]
