CARLSBAD, Calif., July 31, 2023 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the completion of enrollment in the Phase 3 CARDIO-TTRansform cardiovascular outcomes study of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a large and growing patient population at risk of life-threatening cardiovascular (CV) events. The study enrolled more than 1,400 patients. […]
Tag: ATTR-CM
PFIZER CALLS FOR MORE AWARENESS OF TRANSTHYRETIN AMYLOID CARDIOMYOPATHY (ATTR-CM) – A RARE, LIFE-THREATENING DISEASE
KUALA LUMPUR, Malaysia, May 11, 2023 /PRNewswire/ — In an effort to encourage people to take care of their heart health, Pfizer Malaysia is calling for Malaysians to learn more about transthyretin amyloid cardiomyopathy (ATTR-CM), a rare, life-threatening disease. ATTR-CM is caused by unstable transthyretin proteins that misfold and aggregate into amyloid […]
BridgeBio Pharma Presents Updated Results from Phase 2 Open-label Extension Study of Acoramidis in Transthyretin Amyloid Cardiomyopathy (ATTR-CM)
– NT-proBNP, a biomarker of cardiac failure and independent predictor of mortality in ATTR-CM patients, was stable or improving throughout the study. At Month 30, median change from baseline in NT-proBNP was -437 pg/mL with 68% of participants observing NT-proBNP levels below their baseline – Serum TTR levels were sustainably […]
BridgeBio Pharma Reports Month 12 Topline Results from Phase 3 ATTRibute-CM Study
– ATTRibute-CM did not meet its primary endpoint at Month 12. Mean observed six-minute walk distance (6MWD) decline for the acoramidis and placebo arms were 9 meters and 7 meters, respectively. Both declines are similar to healthy elderly adults and less than prior untreated ATTR-CM cohorts – The company observed […]
FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin‑mediated amyloidosis
SILVER SPRING, Md., May 6, 2019 /PRNewswire/ — On May 3, the U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin‑mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Vyndaqel and Vyndamax have the […]
FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin‑mediated amyloidosis
SILVER SPRING, Md., May 6, 2019 /PRNewswire/ — On May 3, the U.S. Food and Drug Administration approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin‑mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Vyndaqel and Vyndamax have the […]