MILAN (ITALPRESS) – The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended marketing authorization for acoramidis, an orally administered selective small molecule that stabilizes transthyretin (TTR), in the European Union (EU). This molecule is indicated for adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive and fatal disease that presents as an infiltrative and restrictive cardiomyopathy leading to heart failure. In vitro, acoramidis has been shown to almost completely stabilize TTR. In the Phase III ATTRibute-CM study, acoramidis showed clear benefits on cardiovascular endpoints.The CHMP’s positive opinion regarding acoramidis is based on the results of the pivotal ATTRibute-CM study. This study evaluated the efficacy and safety of the active ingredient, administered twice daily, compared with placebo in patients with ATTR-CM1. In the Phase III ATTRibute-CM study, acoramidis was shown to be superior to placebo in reducing the composite of all-cause mortality (ACM) and cardiovascular event-related hospitalizations (CVH). “ATTR-CM is often recognized late or misdiagnosed, with negative consequences for patients. It is a condition that, without treatment, progresses inexorably and is ultimately fatal,” said Julian Gillmore, Professor of Medicine at the Centre for Amyloidosis at University College London (UCL), in the United Kingdom. “The positive CHMP opinion represents hope for patients with ATTR-CM. It is encouraging to know that an adjunctive treatment may soon be available that can almost completely stabilize TTR, slowing the progression of symptoms and improving outcomes for these patients. “Patients with ATTR-CM often experience a decline in quality of life due to the physical and functional difficulties associated with their condition. There is a clear need for continued innovation and additional therapeutic options,” said Christine Roth, Executive Vice President, Global Product Strategy and Commercialization and Member of the Pharmaceuticals Leadership Team at Bayer. “The CHMP’s positive recommendation is a milestone in the fight against this serious, life-threatening heart disease. We look forward to European Medicines Agency approval for acoramidis, expected in early 2025, and are working to make this new treatment available to patients as quickly as possible. “The European Commission’s final decision on marketing authorization is expected in the coming months. Acoramidis, developed by BridgeBio Pharma, Inc.(Nasdaq: BBIO), was recently approved by the Food and Drug Administration (FDA) in the United States with an indication that specifies near-complete stabilization of TTR.BridgeBio retains marketing rights in the United States, while Bayer holds exclusive rights for Europe. Pending approval, Bayer plans to launch acoramidis in Europe in the first half of 2025.Since March 2024, Bayer has partnered with BridgeBio and its affiliates for acoramidis. This partnership combines Bayer’s long experience in cardiovascular disease and its strong cardiovascular infrastructure in Europe with BridgeBio’s leadership in the emerging field of ATTR-CM.Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive and fatal disease in older adults characterized by the deposition of abnormal proteins in the heart. It occurs when a tetrameric protein called transthyretin (TTR) becomes unstable, caused by an inherited mutation in the TTR gene or by aging, and dissociates into monomers. These monomers misfold, aggregate, and form amyloid fibrils that are deposited in the heart muscle, eventually leading to heart failure. The disease is often diagnosed late, when amyloid accumulation has already occurred and patients are symptomatic. Once diagnosed, patients with ATTR-CM have a median survival of 3-5 years if untreated.Acoramidis is an orally administered, highly potent, selective transthyretin (TTR) stabilizer designed to mimic a natural “rescue mutation” in the TTR gene (T119M) that targets the root cause of ATTR-CM, destabilization of the native TTR tetramer. In vitro, acoramidis demonstrated almost complete (over 90%) stabilization of TTR. In the Phase III ATTRibute-CM study, acoramidis showed clear benefits on cardiovascular endpoints.In the Phase III ATTRibute-CM study, acoramidis achieved the primary endpoint, demonstrating its superiority over placebo on a hierarchical endpoint that included all-cause mortality (ACM) and cumulative frequency of hospitalizations for cardiovascular events (CVH). Compared with placebo, administered twice daily, acoramidis showed rapid and sustained clinical benefit up to month 30 in patients with ATTR-CM. These are the main results released by Bayer: Significant benefit in the primary endpoint. 36% reduction in the combined endpoint of all-cause mortality or first cardiovascular hospitalization, with benefit evident as early as 3 months of treatment. 50% reduction in cardiovascular hospitalizations. 45% of patients, compared with 9% in the placebo group, showed a reduction (improvement from baseline) in NT-proBNP levels. Forty percent of patients, compared with 22% in the placebo group, showed an increase (improvement over baseline) in distance traveled in 6 minutes.Bayer is a leader in cardiology and is developing a portfolio of innovative treatments for cardiovascular (CV) disease that address unmet medical needs. The company’s strategy is to tap the potential of the future cardiovascular market by transforming its offering of precision cardiology solutions. The goal is to address the growing burden of cardiovascular disease and promote long-term sustainable growth. Bayer’s portfolio already includes several innovative products and molecules currently in various stages of preclinical and clinical development.
– Photo Agency Photogram –
(ITALPRESS).
