Attruby Approved for Transthyretin-Mediated Amyloidosis Cardiomyopathy

On Nov. 22, 2024, BridgeBio Pharma received approval from the US Food and Drug Administration (FDA) for Attruby^™ (acoramidis) to treat cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. Attruby is an oral transthyretin (TTR) stabilizer; the recommended dose is 712mg twice daily. BridgeBio Pharma is expected to launch Attruby soon at an annual cost of approximately $244,000. Full prescribing information can be found here.

At a Glance

• Brand Drug: Attruby^™ (acoramidis)
• Manufacturer: BridgeBio Pharma
• Date Approved: Nov. 22, 2024
• Indication:  for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization
• Dosage Forms Available: 356mg tablets
• Launch Date: In the near future
• Estimated Annual Cost: $244,000
• ATTR-CM is a rare heart condition that is diagnosed in about 6,000 new patients each year in the United States. Some patients inherit the condition, but most cases in the US are spontaneous or “wild type.” Even with a family history of the disease, most cases are not recognized until the patient is an adult.
• The proteins that cause ATTR are folded in ways the body cannot use, so they build up – mainly in the heart and the nerves of the hands, arms, legs, and feet. Amyloid deposits in the heart cause cardiomyopathy that may result in fatigue, shortness of breath, and swelling in the ankles and legs. Atrial fibrillation and heart failure often follow.
• Attruby is in a class of drugs called TTR stabilizers. It works by preserving the function of TTR as a transport protein of thyroxine and vitamin A.
• Approval was based on the Phase III ATTRibute-CM study that demonstrated that Attruby significantly reduced death and cardiovascular-related hospitalization compared to placebo.
• Pfizer’s Vyndaqel^® (tafamidis meglumine) and Vyndamax^® (tafamidis) are oral TTR stabilizers approved in 2019 for treating adults with ATTR-CM. They are taken once daily.
• Alnylam’s Amvuttra^® (vutrisiran) is a transthyretin-directed small interfering RNA medication that was approved in 2022 for treating adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis. It is administered as a subcutaneous injection once every three months. Amvuttra is expected to receive expanded approval for ATTR-CM in April 2025.