Bayer's small molecule TTR stabiliser, acoramidis, has been recommended for approval by the EMA's Committee for Medicinal Products for Human Use (CHMP).
If approved, the orally-administered therapeutic will be used in adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
ATTR-CM is a progressive disease that involves the misfolding and buildup of the transthyretin protein in the heart, which eventually results in heart failure.
The CHMP's recommendation is based on the results of the Phase III ATTRibute-CM study, which found that acoramidis can reduce the risk of all-cause mortality and ATTR-CM-related hospitalisations, while also enhancing the 6 minute walk distance after 30 months of treatment.
In vitro studies have also revealed that acoramidis can stabilise TTR by ≥90%.
Moving forward
Following the CHMP's verdict, Bayer plans to submit a marketing authorisation application to the UK's regulatory board for the same indication.
“ATTR-CM is often delayed in its recognition or is misdiagnosed, which can have a negative impact on patients’ outcomes. It is a condition that progresses in the absence of treatment and is ultimately fatal,” said Julian Gillmore, Professor of Medicine, University College London (UCL) Centre for Amyloidosis, UK.
“The CHMP's positive opinion offers hope to individuals living with ATTR-CM. We are encouraged by the prospect of additional treatment to slow the progression of symptoms and improve outcomes in patients with ATTR-CM.”
The European Commission's final decision will be made within the coming months; the drug has already been approved by the FDA, though BridgeBio holds the marketing rights for acoramidis in this region.
Bayer's Executive VP of Global Product Strategy and Commercialisation, Christine Roth, commented: “Patients living with ATTR-CM often see a decline in their quality of life due to the physical and functional challenges posed by their condition. There is a clear need for continued innovation and further options for patients affected,”
"This positive recommendation from the CHMP represents a significant milestone in the fight against this life-threatening heart disease.”