ATTRUBY is a newly approved medication offering hope for adults with cardiomyopathy caused by transthyretin-mediated amyloidosis (ATTR-CM). It aims to reduce cardiovascular death and hospitalizations in patients with this rare and challenging condition. ATTRUBY was approved on November 22, 2024.
What is the name of the drug, and what does it do?
ATTRUBY (pronounced ah-troo-be) is a transthyretin stabilizer. It works by binding to the transthyretin protein (TTR) and preventing it from breaking down into smaller pieces that form amyloid deposits in the heart. This action helps to stabilize heart function and slow disease progression.
How does it work?
ATTRUBY specifically targets TTR at key binding sites, slowing the process that leads to amyloid buildup. By doing this, it helps maintain the structure of the TTR protein and reduces the damage caused by amyloid deposits. This helps improve heart function and lowers the risk of heart-related complications.
What did the research discover?
In a 30-month clinical trial involving 611 participants with ATTR-CM, researchers found that ATTRUBY provided significant benefits. Patients treated with ATTRUBY had a 35.5% lower risk of cardiovascular-related hospitalizations or death compared to those on a placebo. The medication also improved patients’ physical endurance, as measured by a six-minute walk test, with an average improvement of 40 meters compared to placebo. Additionally, it enhanced health-related quality of life, as demonstrated by a notable increase in Kansas City Cardiomyopathy Questionnaire scores. These results show that ATTRUBY can slow disease progression and improve patients’ quality of life.
What are some of the side effects?
- Stomach-area (abdominal) pain
- Diarrhea
These side effects were mild and resolved without stopping treatment in most cases. Laboratory tests also showed temporary changes in kidney function that returned to normal after stopping the medication.
What are the dosage recommendations, and how is it prescribed?
ATTRUBY is taken as two tablets (356 mg each) twice daily, with or without food. Patients should swallow the tablets whole and avoid cutting, crushing, or chewing them. Consistent use is key to achieving the best results.
Source
BridgeBio Pharma, Inc. Prescribing information for ATTRUBY (acoramidis). November 2024. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/216540s000lbl.pdf
