BridgeBio submits NDA for Transthyretin Amyloid Cardiomyopathy (ATTR-CM) candidate

 - NDA submission is based on positive results from ATTRibute-CM Phase 3 study, including a highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001), on the primary endpoint (a hierarchical analysis prioritizing in order all-cause mortality (ACM), then frequency of cardiovascular-related hospitalization (CVH), then change from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP), then change from baseline in 6-minute walk distance (6MWD))

- In ATTRibute-CM, acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual CVH frequency, as well as improvements for a large proportion of patients on laboratory and functional measures

- ATTRibute-CM results also demonstrated rapid clinical benefit on the composite endpoint of ACM and CVH in patients treated with acoramidis

- Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified

https://www.biospace.com/article/releases/bridgebio-pharma-announces-submission-of-new-drug-application-nda-to-u-s-food-and-drug-administration-fda-for-acoramidis-for-the-treatment-of-patients-with-transthyretin-amyloid-cardiomyopathy-attr-cm-/