European Commission Approves BridgeBio Treatment for ATTR-CM
Acoramidis was approved by FDA in November 2024 and is prescribed in the US under the brand name Attruby.
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BridgeBio Pharma, of Palo Alto, Calif., has announced the European Commission (EC)’s marketing authorization approval for Beyonttra (acoramidis), for treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) (1) in the European Union. In a press release on Feb. 11, 2025, BridgeBio said the treatment is the first to be approved with a label specifying a near-complete (≥90%) transthyretin (TTR) stabilizer.
According to BridgeBio, information published in JAMA Cardiology in December 2024 suggested that relative increases in serum TTR concentrations, as the result of greater TTR stability, are linked to a reduced risk of both all-cause and specific cardiovascular mortality in the general population (1).
A Phase III study (ATTRibute-CM) on which EC based its approval showed the most rapid benefit of any such study of acoramidis to date, BridgeBio said. This included the time to first event—meaning all-cause mortality or cardiovascular-related hospitalization (CVH)—durably separated, relative to placebo, in as few as three months, and up to a 50% reduction in cumulative frequency of CVH events relative to placebo at month 30 (1).
BridgeBio’s acoramidis treatment was previously among five drugs recommended for orphan drug status in December 2024 by the European Medicines Agency’s Committee for Medicinal Products for Human Use as part of the committee’s larger recommendation of 17 drugs for market authorization, also including six biosimilar recommendations (2).
“ATTR-CM is a rapidly progressing disease with a poor prognosis when left untreated, making the approval of acoramidis, which has demonstrated improved benefit on all-cause mortality and cardiovascular-related hospitalizations in as few as three months, a very important accomplishment for patients. We are pleased that people living with ATTR-CM will have access to another treatment option in the EU,” said Marianna Fontana, MD, PhD, professor of cardiology and honorary consultant cardiologist at the National Amyloidosis Centre, Division of Medicine, University College London, in the BridgeBio press release (1).
BridgeBio has been collaborating on acoramidis since March 2024 with Bayer, which will be responsible for all commercial activity for the treatment in the EU, according to the press release (1). With this approval, BridgeBio will receive a $75 million milestone payment from Bayer, and also will be due royalties in a tiered structure.
“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,” said Jonathan Fox, MD, PhD, BridgeBio cardiorenal chief medical officer, in the release. “This approval would not have been possible without the commitment of the clinical trial participants and their families, and the dedicated support of the physicians and scientists involved in the clinical program. Alongside our able partners at Bayer, we look forward to this new opportunity to serve ATTR-CM patients across the European Union.”
The EC authorization of BridgeBio’s product comes one week short of exactly five years since Pfizer’s Vyndaqel (tafamidis) was approved by the same body on Feb. 18, 2020, making it the first available treatment for patients with ATTR-CM in the EU (3). In March 2022, AstraZeneca’s Alexion rare disease group announced an exclusive partnership with Neuroimmune to develop, manufacture, and commercialize NI006, at the time an investigational treatment for ATTR-CM (4).
Acoramidis will be launched by Bayer in the EU in the first half of 2025 (1). The treatment is currently also under review by both the Japanese Pharmaceuticals and Medical Devices Agency and the Brazilian Health Regulatory Agency.
References
1. BridgeBio Pharma. Beyonttra (acoramidis), The First Near Complete TTR Stabilizer (≥90%), Approved by the European Commission to Treat ATTR-CM. Press Release. Feb. 11, 2025.
2. EMA. Meeting Highlights from the Committee for Medicinal Products for Human Use (CHMP) 9–12 December 2024. Press Release. Dec. 13, 2024.
3. Pfizer. European Commission Approves Vyndaqel, The First Treatment in the EU for Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Press Release. Feb. 18, 2020.
4. AstraZeneca and Neurimmune Close Exclusive Global Collaboration and Licence Agreement to Develop and Commercialise NI006. Press Release. March 1, 2022.
