Acoramidis

In its final analysis of Pfizer Inc. 's widely used drug for transthyroid amyloid cardiomyopathy (ATRCM), the U.S. drug cost regulator called for a reduction of at least 85 percent from its current list price of nearly $268,000 a year to meet generally accepted cost-effectiveness standards. The conclusion, made official in the latest ATRCM report released Monday by the Institute for Clinical and Economic Review (ICER), makes clear that Pfizer's two tafetamines, Vyndaqel and Vyndamax, are subject to the discount requirement. According to ICER's data analysis, in order to meet the cost-effectiveness criteria of between $100,000 and $150,000 per quality-adjusted life year (QALY) or equivalent life year acquired, the annual cost of Tarfamides and the thyrotropin stabilizer category to which it belongs should be adjusted to between $13,600 and $39,000. That means a price cut of between 85 and 95 per cent. The proposed annual cost range is revised up from a preliminary draft report in July. In its response, Pfizer reiterated the challenges and biases it faces in using QALEs as a cost-effectiveness measure for rare diseases, particularly for older patients who have a shorter life expectancy and limited time to benefit from treatment. In addition, the report comes at a time when the FDA is about to make a decision on BridgeBio Pharmaceuticals' competing drug acoramidis, and Alnylam Pharmaceuticals recently submitted an application for its RNA silencer Amvuttra (vutrisiran). This could further affect the competitive landscape of the ATRCM market. It is worth noting that ICER's price assessment covered Tafamidi and acoramidis, but did not include Amvuttra. Although Amvuttra was approved by the FDA for the treatment of ATR polyneuropathy in 2022 and has an annual price tag of $463,500, ICER said there is still not enough data to build an economic model for it. ICER's chief medical officer, Dr. David Rind, noted that the reasons for the price range adjustment include a change in the mortality estimate methodology and the correction of a calculation error in the previous quality of life assessment. At the same time, he highlighted the difficulty in economic evaluation of Amvuttra, especially the lack of data on the prediction of disease change in patients with NYHA Class III heart failure. However, he expects that the information may eventually be made public to meet the requirements of the European health assessment. In the final report, ICER also raised the complexity of comparing the net health benefits of Amvuttra versus stabilizers, noting that the question of the advantages of combination therapy versus monotherapy needs further study. To that end, the group recommends that researchers work to build a comparative profile of the three drugs and stresses the importance of randomized clinical trials to answer this question.

PALO ALTO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, announced today that outcomes data through 42 months from the ongoing long-term open-label extension of ATTRibute-CM, its Phase 3 study of acoramidis in ATTR-CM, will be shared in a featured science oral presentation at the American Heart Association (AHA) Scientific Sessions, taking place in Chicago, Illinois on November 16 – 18, 2024. In addition to the presentation, BridgeBio was selected to share three posters in moderated poster sessions on ATTR-CM. Featured Science Oral Presentation:Acoramidis Reduces All-Cause Mortality (ACM) and Cardiovascular-Related Hospitalization (CVH): Initial Outcomes from the ATTRibute-CM Open-Label Extension (OLE) StudySession Title: Featured Science: Amyloid, Hypertrophic, and Danon Cardiomyopathies: Targeted Therapies and Specific PopulationsPresenter: Daniel Judge, M.D. of Medical University of South CarolinaDate: Monday, November 18 at 9:45 am CT/10:45 am ET Moderated Digital Posters:Costs and Healthcare Resource Utilization in Transthyretin Amyloid Cardiomyopathy Exceeds That of Generalized Heart Failure Session Title: Socioeconomic Insights and Innovations in Heart FailurePresenter: Justin Grodin, M.D. of UT Southwestern Medical CenterDate: Sunday, November 17 at 10:00 am CT/11:00 am ET Evolving Baseline Risk in Patients with Transthyretin Amyloid Cardiomyopathy: A Systematic Literature Review of Clinical Trials Session Title: Cardiac Amyloidosis 2024: Advances in Prognostication and Management Presenter: Ahmad Masri, M.D. of Oregon Health & Science UniversityDate: Sunday, November 17 at 11:10 am CT/12:10 pm ET Acoramidis Improved Survival in Patients with Transthyretin Cardiac Amyloidosis Regardless of Prior Cardiovascular Hospitalization Session Title: Cardiac Amyloidosis 2024: Advances in Prognostication and Management Presenter: Kevin Alexander, M.D. of Stanford University School of MedicineDate: Sunday, November 17 at 11:20 am CT/12:20 pm ET The presentation and posters will be available following the sessions on the Presentations page within the Investors section of the BridgeBio website at https://investor.bridgebio.com. About BridgeBio Pharma, Inc.BridgeBio Pharma (BridgeBio) is a commercial-stage 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 bridgebio.com and follow us on LinkedIn, Twitter and Facebook. BridgeBio Contact:Vikram Balicontact@bridgebio.com(650)-789-8220