The U.S. Food and Drug Administration (FDA) has approved Acoramidis (brand name Attruby), marking it as the first “near-complete” stabilizer for treating transthyretin amyloid cardiomyopathy (ATTR-CM). This approval, announced late Friday, is based on the findings from the ATTRibute-CM clinical trial, which demonstrated significant cardiovascular benefits for patients.
Acoramidis is now the second medication approved for ATTR-CM, following Pfizer’s tafamidis, which was previously criticized for its high annual cost of $268,000. The price for Acoramidis is set at approximately $244,000 per year, raising concerns about affordability for patients outside of clinical trials. BridgeBio Pharma, the manufacturer of Acoramidis, has pledged to provide the drug free of charge for life to participants in the trial.
The ATTRibute-CM trial involved 632 patients and showed that those taking Acoramidis at a dosage of 800 mg twice daily experienced improvements in key health metrics over 30 months. These included reductions in all-cause mortality and cardiovascular-related hospitalizations, with outcomes approaching those of age-matched individuals without the condition.
Dr. Martha Grogan from the Mayo Clinic emphasized the significance of this new treatment option, stating that it offers hope for better management of a disease that was once considered fatal. The trial results indicated that Acoramidis not only stabilizes transthyretin but also improves overall quality of life for patients.
BridgeBio plans to seek regulatory approval for Acoramidis in Europe, Japan, and Brazil. Additionally, there are emerging therapies in development; a recent presentation at the American Heart Association’s Scientific Sessions highlighted promising results from a gene-editing therapy targeting the TTR gene.
As discussions around drug pricing continue, a report from the Institute for Clinical and Economic Review suggested that transthyretin-stabilizing agents should ideally cost between $13,600 and $39,000 annually to meet cost-effectiveness standards. This would require substantial reductions in current list prices.
Related topics:
- HMS Study Reveals Connection Between Heart Condition And Cancer Treatment
- Long COVID Inflammation Linked to Heart Cell Damage
- SpaceX CRS-31 Mission to Enhance Heart Health, Neurodegeneration Research, And Student Science Initiatives