Approval Granted for Acoramidis in Treating Rare Heart Condition

Amyloidosis encompasses a group of rare diseases characterized by the deposition of misfolded proteins, which can affect various organs and tissues in the body. One notable form is Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM).

The accumulation of these misfolded proteins can lead to severe organ dysfunction. In cases where the heart is affected, amyloidosis may result in serious complications, including cardiomyopathy and heart failure.

The underlying cause of cardiac amyloidosis is often linked to the protein transthyretin (TTR). In ATTR-CM, the TTR tetramer dissociates into its monomers, which misfold and aggregate into oligomeric amyloid precursors. These precursors deposit in the heart, forming amyloid fibrils that cause stiffening of the cardiac muscle, thus impairing its ability to pump blood effectively.

The newly approved medication, Acoramidis (marketed as Beyonttra(TM)), is a TTR stabilizer similar to Tafamidis, which has been approved for a different indication. Acoramidis interacts with neighboring serine residues within the TTR tetramer's thyroxine-binding sites, creating hydrogen bonds that mimic a protective mutation of TTR. This interaction enhances the stability of the tetramer, prevents its dissociation into monomers, and subsequently slows the amyloidogenic process that leads to ATTR-CM.

The approval of Acoramidis was supported by data from the Phase III clinical trial known as ATTRibute-CM. This placebo-controlled study involved 632 participants diagnosed with symptomatic ATTR-CM, assessing the efficacy and safety of Acoramidis. Results indicated that the treatment significantly reduced the rate of cardiovascular-related hospitalizations, improved survival rates, and maintained patients' functional capacity and quality of life over a 30-month period.

The recommended dosage for Acoramidis is 712 mg, administered twice daily (two 356 mg film-coated tablets). The trial noted that the most common side effects reported were diarrhea (12%) and gout (11%).

According to the marketing authorization holder, Bayer will oversee all commercial activities related to Beyonttra in the European Union, with the medication expected to become available in the market in the first half of 2025. In the United States, Acoramidis is already available under the brand name Attruby(TM).