The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has endorsed acoramidis for market authorization in the European Union. Acoramidis is an orally consumed, selective small-molecule stabilizer of transthyretin (TTR). It targets adult individuals diagnosed with transthyretin amyloid cardiomyopathy (ATTR-CM), a serious progressive condition characterized by restrictive cardiac issues that lead to heart failure. Laboratory results indicate that acoramidis can almost entirely stabilize TTR. Strong positive outcomes in cardiovascular measures were observed during the pivotal phase III ATTRibute-CM trial.
The CHMP’s advice is founded on the favorable findings from the ATTRibute-CM trial, which evaluated acoramidis’ effectiveness and safety against a placebo in patients with ATTR-CM. The study showed that acoramidis outperformed the placebo in lowering the combined results of mortality from any cause and hospitalizations related to cardiovascular issues.
“ATT-CM is a condition frequently misdiagnosed or recognized late, severely impacting patients’ well-being. With treatment, it’s crucial to slow the progression and associated mortality,” commented Professor Julian Gillmore from UCL’s Centre for Amyloidosis. “The positive opinion from CHMP presents hope for those enduring ATT-CM. Introducing acoramidis has the potential to meaningfully halt symptom advancement and improve patient outcomes.”
“Living with ATT-CM poses numerous physical and daily-life hurdles, necessitating further innovation in treatments,” stated Christine Roth from Bayer. “CHMP’s positive stance is an essential breakthrough against this dangerous heart disorder. We anticipate EMA’s approval by early 2025 and aim to make acoramidis accessible to patients promptly.”
The EU’s final decision is awaited from the European Commission. BridgeBio Pharma, Inc., which developed acoramidis, has secured FDA approval in the US with specifications for high TTR stabilization. While BridgeBio holds US marketing rights, Bayer is responsible for marketing in Europe, with plans to introduce acoramidis on the continent by mid-2025.
Since March 2024, a collaboration emerged between Bayer and BridgeBio, merging Bayer’s expertise in cardiovascular conditions with BridgeBio’s pioneering role in ATT-CM treatment.
ATT-CM is prevalent in older adults and results from abnormal heart protein buildup. The root cause is unstable TTR owing to genetic mutations or aging, leading to heart function impairment. Typically diagnosed late, untreated patients have a three to five-year survival outlook.
Acoramidis acts as a potential remedy by mimicking a beneficial genetic variant to stabilize the original TTR configuration. Tests affirm its tremendous stabilization capacity, and clinical trial results corroborate it as a superior option against placebo on predefined health benchmarks.
Bayer, renowned in cardiology, continues to expand its cardiovascular solutions in areas of significant unmet need, aiming for substantial growth by focusing on precise cardiology applications due to significant cardiovascular disease impacts.