BridgeBio Pharma's Attruby™ Approved by FDA for Treating Transthyretin Amyloid Cardiomyopathy

BridgeBio Pharma, Inc., a biopharmaceutical company pioneering treatments for genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Attruby™ (acoramidis) orally-administered near-complete (≥90%) stabilizer of Transthyretin (TTR) is designed to treat adults with Transthyretin amyloid cardiomyopathy (ATTR-CM), aiming to reduce cardiovascular death and cardiovascular-related hospitalization. The FDA approval is based on positive outcomes from the ATTRibute-CM Phase 3 study.

Publish Date: 22-11-2024 Source: BridgeBio Pharma, Inc.

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a form of systemic amyloidosis characterized by misfolded transthyretin (TTR) protein deposition in the myocardium. Another notable cause of cardiac amyloidosis is the accumulation of immunoglobulin light-chain (AL) aggregates. Although several amyloidogenic proteins may be deposited in various organs and tissues, myocardial involvement is rare. Symptoms of ATTR-CM can vary, and the condition is frequently misdiagnosed, with early stages resembling symptoms of other conditions such as hypertensive heart failure or hypertrophic cardiomyopathy. Some patients may be asymptomatic, while others may progress to end-stage heart failure. The disease is classified based on the sequence of the TTR gene, distinguishing between wtATTR-CM (no mutation) and hATTR-CM (presence of a mutation). In 2019, the FDA approved tafamidis and tafamidis meglumine for treating ATTR-CM. Both agents selectively bind to the transthyretin tetramer, preventing the destabilization of the transthyretin transport protein and the subsequent amyloid formation that leads to ATTR-CM.

The estimated incidence rate of ATTR-CM is 3.6 cases per 100,000 persons annually in the United States.

However, the current Transthyretin Amyloid Cardiomyopathy (ATTR-CM) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (NI006, ALXN2220, AT-01, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Transthyretin Amyloid Cardiomyopathy (ATTR-CM) treatment. The key companies in the advanced development stage are Neurimmune AG, Alexion Pharmaceuticals, Inc., Attralus, Inc., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Transthyretin Amyloid Cardiomyopathy (ATTR-CM) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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