FDA Approves Attruby (Acoramidis) for ATTR-CM, Offering New Hope for Patients with Transthyretin Cardiac Amyloidosis

FDA Approval:
The U.S. Food and Drug Administration (FDA) has approved Attruby (acoramidis), a near-complete TTR stabilizer (≥90%), for the treatment of adults with transthyretin cardiac amyloidosis (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization[1][2].

Clinical Trial Results:
The approval is based on the positive results from the ATTRibute-CM Phase 3 study, which showed that Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life in patients with ATTR-CM[1][2].

Mechanism of Action:
Attruby was designed to mimic a naturally occurring “rescue mutation” of the TTR gene (T119M) that targets the root cause of ATTR-CM, the destabilization of the native TTR tetramer. It preserves the native function of TTR as a transport protein of thyroxine and vitamin A and demonstrates benefit on cardiovascular outcomes[1][2].

Patient Support:
BridgeBio offers a patient support services program, ForgingBridges, to help patients and their families access this new therapy, including insurance resources, financial assistance options, and a dedicated support team[1].

Global Expansion:
BridgeBio has submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025, and has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe[1][2].

Pricing and Accessibility:
The price of Attruby is expected to be $18,759.12 for a 28-day supply. BridgeBio will provide Attruby free for life to clinical trial participants[2].

Market Competition:
The approval sets up competition with Pfizer and Alnylam in the treatment of ATTR-CM[3].