Approvals

The European Commission (EC) has approved acoramidis to treat wild-type or variant transthyretin amyloidosis in adults with cardiomyopathy (ATTR-CM), according to an announcement from the drug’s developer BridgeBio Pharma.

BridgeBio is now set to receive a $75m milestone payment from Bayer, which has an exclusive licence to commercialise the drug for ATTR-CM in Europe.

Affecting approximately 400,000 people globally, ATTR-CM is a progressive condition that occurs when the transthyretin (TTR) protein becomes misshapen and builds up in the heart. These deposits can lead to heart failure, making it difficult for the heart to pump blood properly.

Acoramidis, which will be marketed in the EU under the brand name Beyonttra, is designed to mimic a naturally occurring rescue mutation of the TTR gene that targets the root cause of ATTR-CM and is now the only product approved by the EC with a label specifying near-complete TTR stabilisation.

The regulator’s decision on the drug follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by positive results from the late-stage ATTRibute-CM trial of 632 patients with symptomatic ATTR-CM.

Results showed that acoramidis significantly improved survival and reduced cardiovascular-related hospitalisation after 30 months of treatment compared to placebo.

Jonathan Fox, BridgeBio cardiorenal chief medical officer, said: “The EU approval of acoramidis is a significant advancement for patients living with ATTR-CM in need of new disease-modifying treatments for their condition… Alongside our able partners at Bayer we look forward to this new opportunity to serve ATTR-CM patients across the EU.”