The US Food and Drug Administration (FDA) has approved BridgeBio Pharma’s Attruby (acoramidis) to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM).
The orally-administered drug has been specifically authorised to reduce cardiovascular death and cardiovascular-related hospitalisation in patients with the rare heart disease.
Affecting up to 500,000 people worldwide, 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.
Attruby is designed to mimic a naturally occurring “rescue mutation” of the TTR gene that targets the root cause of ATTR-CM and, following the FDA’s approval, Attruby is now the first and only approved product with a label specifying near-complete stabilisation of TTR.
The US regulator’s decision on the drug was supported by positive results from the late-stage ATTRibute-CM trial of 632 patients with symptomatic ATTR-CM.
Results showed that Attruby significantly improved survival and reduced cardiovascular-related hospitalisation after 30 months of treatment compared to placebo.
Kristen Hsu, executive director of research at the Amyloidosis Research Consortium, described the authorisation as a “significant milestone” for patients.
“This new therapy provides more choices for patients and healthcare providers, helping to better manage this challenging disease. We’re hopeful that Attruby will improve outcomes and bring greater hope to those living with ATTR-CM,” she said.
BridgeBio’s founder and chief executive officer, Neil Kumar, said the company’s “journey [with Attruby] is not over as [it looks] to pursue approvals globally, next in Europe, Japan and Brazil, and to continue exploring the full potential of this treatment”.
Bayer has an exclusive licence to commercialise acoramidis for ATTR-CM in Europe, while AstraZeneca’s rare disease unit Alexion maintains an exclusive licence for the asset in Japan.
Earlier this year, AstraZeneca shared positive phase 3 results that showed consistency with those in ATTRibute-CM, including survival, cardiac-related hospitalisations and other measures of improved functions and quality of life at 30 months.
