A treatment for cardiac amyloidosis arrives in Spain

Bayer announced this Monday the availability in Spain of Beyonttra (acoramidis), the first treatment that offers a practically complete stabilization of transthyretin (TTR) in patients with cardiac transthyretin amyloidosis (ATTR-CM), both native and variant types.

Beyonttra is a small, selective, orally administered molecule designed to act on the origin of the disease by reducing amyloid deposits in the heart and slowing its progression, Bayer reported.

The ATTR-CM is a serious pathologyunderdiagnosed and undertreated, produced by the destabilization of the TTR protein, essential in functions such as the transport of thyroxine and vitamin A. Its unfolding generates amyloid fibrils that alter the structure and function of the heart, which can lead to heart failure, recurrent hospitalizations, functional deterioration and premature mortality. The average survival without treatment is between 2.6 and 3.6 years.

The disease could affect more than 400,000 people globallymostly still undiagnosed, due to nonspecific symptoms such as fatigue, dyspnea or edema. Patients wait an average of 39 months to receive a correct diagnosis, and up to 60% consult with three or more doctors before reaching a definitive diagnosis.

Despite recent advances, challenges persist in the clinical and therapeutic management of ATTR-CM. According to the doctor Pablo García-Pavíahead of the Family Heart Disease Unit of the Puerta de Hierro Hospital Majadahonda“the availability of a new alternative that almost completely stabilizes TTR, helping to reduce the production of amyloid fibrils, marks a relevant advance in clinical practice. This new therapy allows intervention in earlier stages of the pathological process, being able to offer patients more time to live and more time out of the hospital, opening new perspectives in the management of ATTR-CM.”

Beyonttra acts as selective oral modulatorpractically completely stabilizing (≥90%) TTR, inspired by a protective natural mutation, T119M, observed in some people, achieving greater natural stability of TTR. It is the only stabilizer with this level of stabilization according to the technical sheet. Its mechanism prevents the unfolding of TTR and the formation of amyloid fibrils, preserving cardiac function and prolonging survival.

The doctor Rocío Eiros Bachelorcardiologist at the University Hospital of Salamanca, highlights that clinical data show “rapid differences compared to placebo in quality of life variables and key biomarkers, in addition to excellent, and also rapid, results in mortality and cardiovascular hospitalization. This new therapy offers a real opportunity to modify the course of the disease, with quickly measurable and observable results.”

The approval is supported by the phase III ATTRibute-CM trial, which evaluated acoramidis versus placebo in patients with wild-type or variant ATTR-CM. The results showed that treated patients had an 80% greater probability of achieving a better overall clinical outcome, as well as a 36% reduction in the combined risk of all-cause mortality or first cardiovascular hospitalization at 30 months. Treatment also reduced the annualized rate of cardiovascular hospitalizations by 50%, with additional improvements in functional capacity and quality of life, and virtually complete stabilization of TTR. In addition, it has shown a long-term safety profile.

The medical director of Bayer Spain, doctor Guido Senatore, underlines that this launch represents “one more step in that trajectory, by offering a pioneering solution in a highly complex disease where until now the options were very limited.