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2025 Spring NewsletterMay 21, 2025

Be sure to catch up on the latest happenings in our fall newsletter here!

Pfizer Mounts Defense of Cardiac Blockbuster as Alnylam, BridgeBio Enter MarketMay 21, 2025

Alnylam and BridgeBio are competing for people who are switching from Pfizer’s blockbuster ATTR amyloidosis drug tafamidis while all three companies are fighting for new patients.
A three-way tussle has emerged in the growing ATTR amyloidosis market as Alnylam and BridgeBio Pharma have secured FDA approvals to challenge Pfizer.


Tafamidis, which Pfizer sells as Vyndaqel, Vyndamax and Vynmac, had the ATTR...

Plain Language Summary of Publications (PLSPs)May 21, 2025

Below is a resource for our patients:

Taylor & Francis is a publisher of peer-reviewed medical journals. We publish a type of article called Plain Language Summary of Publications (PLSPs). These articles provide an overview of a published, scientific journal article written in non-technical language. These summaries enable a wide audience, including patient audiences, to be able to access and...

First Participant Dosed with Acoramidis in ACT-EARLY, the First Ever ATTR Primary Prevention StudyMay 14, 2025

– ATTRibute-CM, BridgeBio’s Phase 3 clinical trial of acoramidis in patients with ATTR-CM, achieved statistical significance in reducing the risk of ACM or first CVH versus placebo in ATTRv-CM patients (59.1% risk reduction), establishing the mechanistic hypothesis that stabilizing TTR may delay or prevent ATTRv-CM

– ACT-EARLY is a registrational, randomized, double blind, placebo controlled, event driven prevention study that...

Acoramidis Shows Statistically Significant Improvements in Cardiovascular Outcomes in Patients with Variant ATTR-CMApril 3, 2025

  • Acoramidis’s hazard ratio of .41 for time to ACM or first CVH versus placebo in the ATTRibute-CM study subgroup of ATTRv-CM patients achieved statistical significance in a pre-specified analysis

     
  • This profound treatment effect, due to the near-complete stabilization (≥90%) and binding of TTR, represents the greatest observed benefit to date for ATTRv-CM patients, an ATTR-CM population with poor...