American Heart Association

FDA accepts Alnylam's application to expand Amvuttra's label for transthyretin amyloid cardiomyopathy, potentially challenging Pfizer's tafamidis. Amvuttra, an RNAi therapeutic, aims to be the first RNAi treatment for ATTR-CM, supported by Phase III HELIOS-B trial results showing reduced mortality and CV events. FDA sets PDUFA date for March 23, 2025.

PIONEER, an AI-powered tool, predicts human protein interactions, aiding in cancer research and precision medicine by analyzing mutations and treatment responses. Developed by Haiyuan Yu and Feixiong Cheng, it extends AlphaFold's capabilities to complex protein-to-protein interactions, with potential applications in various disorders.

A new AI tool, PIONEER, predicts protein interactions, aiding research in precision medicine and therapy development. Applied to 33 cancer types, it predicts drug responses and patient survival rates. Available as a web platform and software, it also supports research in model organisms like mice. Developed by Haiyuan Yu and Feixiong Cheng, it builds on AlphaFold, enhancing understanding of protein-to-protein interactions.

The FDA approved acoramidis (Attruby), an oral transthyretin stabilizer for adults with ATTR-CM, reducing cardiovascular death and hospitalization. This first-of-its-kind therapy, designed to mimic a natural TTR gene mutation, was supported by the ATTRibute-CM trial, showing improved outcomes in ATTR-CM patients.

New stroke prevention guidelines from the American Heart Association and American Stroke Association focus on high-risk groups, including women with high blood pressure during pregnancy or early menopause, and transgender women on hormone therapy. The guidelines recommend aggressive blood pressure control, a Mediterranean diet, and GLP-1 weight-loss drugs for diabetes patients. They also emphasize social determinants of health and the American Heart Association's Life's Essential 8 for stroke prevention.

Cardiol Therapeutics' CEO, David Elsley, discusses CardiolRx™, an oral therapy for recurrent pericarditis and acute myocarditis, highlighting Phase 2 trial results showing pain reduction and decreased inflammation. Upcoming Phase 2/3 trial results expected in 2025 could expedite market entry under FDA's orphan drug designation.

Despite ACA progress, 8% of Americans remain uninsured, and 25% are underinsured, facing high costs and delayed care. The FDA's approval of isatuximab highlights underrepresentation of Black patients in clinical trials. An American Board of Cardiovascular Medicine is proposed to address care complexities. Racial disparities in breast cancer care among older adults are significant, with Black patients facing higher mortality rates. Global Biosimilars Week discusses challenges in biosimilar adoption, calling for policy reforms to improve access.

TTHealthWatch discusses genetic testing for couples planning pregnancy, use of two medications post-heart attack, genome-wide association models for cardiovascular disease risk, and two strategies to reduce lipoprotein(a). Polygenic risk scores show limited individual precision, and nationwide genetic carrier screening in Australia identified 1.9% of couples at risk for genetic conditions, with 77% opting for reproductive interventions.

Polygenic risk scores (PRS) improve prediction accuracy of complex traits and diseases, with ethical and implementation challenges discussed. PRS use may exacerbate health disparities, necessitating new ethical perspectives and clinical guidelines. Realistic expectations and patient-provider communication are crucial for effective PRS application.

Arrowhead Pharmaceuticals submitted an FDA application for plozasiran, targeting familial chylomicronemia syndrome (FCS), with no current FDA-approved treatments. The drug showed sustained reductions in apolipoprotein C-III and triglycerides, significantly lowering pancreatitis risk. The FDA granted breakthrough therapy designation in 2024.