
A new drug for the treatment of a rare heart disease has been approved in the USA
The U.S. Food and Drug Administration (FDA) has approved a drug for the treatment of progressive genetic heart disease – ATTR-CM.
The drug Atturby (acoramidis) has shown its effectiveness in phase III clinical trials involving 632 patients. After 30 months of taking the study drug, there was a 42% decrease in overall mortality and the number of hospitalizations associated with cardiovascular diseases compared with placebo.
The active pharmaceutical substance of Atturby, acoramidis, provides almost complete (more than 90%) stabilization of the protein transtiretin (TTR), which transports thyroid hormone thyroxine and vitamin A throughout the body. This protein is a complex of four subunits. Due to age-related changes or genetic mutations, TTR can break down into separate parts, which can fold incorrectly and form toxic amyloid fibrils, leading to the development of cardiomyopathy and heart failure.
Currently, tafamidis preparations manufactured by Pfizer are used for the treatment of ATTR-CM. In the Russian market, these drugs are presented under the trade names Vindamex (registration date 07/12/2024) and Vindakel (registration date 04/13/2022). In Russia, these drugs have orphan status.
Tafamidis is also a stabilizer of the transthyretin protein and binds to it in thyroxine binding sites, stabilizing the four-component structure of the protein and slowing its breakdown.
According to the developer of the Atturby drug, BridgeBio, tafamidis drugs provide only 50% stabilization of TTR, and clinical research data indicate that tafamidis reduces the number of deaths and hospitalizations associated with heart disease by about 30%, which indicates a higher efficacy of the Atturby drug.
The European Union is also considering an application for registration of Atturby. The rights to this drug in the EU belong to Bayer, which will pay BridgeBio up to $ 310 million if the drug is approved by the regulator.