Transthyretin amyloidosis (ATTR) is a rare, life‐threatening disease resulting from aggregation and deposition of a defective type of protein called (‘amyloids’) in various tissues. These deposits damage the structure and function of the tissues and cause serious disease which is usually fatal if it affects major organs. Transthyretin amyloidosis cardiomyopathy (ATTR‐CM) primarily affects the heart, causing thickening and stiffening of the heart tissues. Symptoms usually start after age 60 years and include shortness of breath, sometimes after only mild exertion; palpitations and abnormal heart rhythms, most frequently atrial fibrillation or atrial flutter; ankle swelling (oedema); fatigue; fainting, and angina (chest pain). ATTR‐CM is progressive and ultimately leads to death. Current treatment focuses mainly on managing the symptoms although heart transplantation may be appropriate for some patients.
Tafamidis is an oral (taken by mouth) drug that has the potential to slow the formation of the amyloid deposits that can produce heart problems in people with ATTR‐CM. Tafamidis works by specifically interfering with the disease process that leads to the formation and deposition of the amyloids within the heart tissues. If approved, tafamidis will become the first medicinal treatment option specifically indicated for treating ATTR‐CM, in patients that currently have very limited options available and that mostly focus on symptom management.
Rivaroxaban is an oral medicine which prevents the development of blood clots by blocking the formation of a molecule called thrombin which is a key part of the process of blood clot formation. Rivaroxaban has the potential to reduce thrombin and blood clot formation in people with heart failure and coronary artery disease. This is potentially important as patients with heart failure after an episode of acute decompensation have been seen to have increased levels of thrombin. No other drug is currently licenced for use in this group of patients which targets thrombin.