Acoramidis Approval Signals Expanding Small Molecule Pipeline for ATTR-CM

BridgeBio Pharma's ATTRUBY (acoramidis) entered the market on November 22, 2024, following FDA approval for the treatment of cardiomyopathy caused by wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults. The approval adds a twice-daily oral transthyretin stabilizer to a therapeutic category where manufacturing complexity and regulatory scrutiny of solid oral dosage forms are increasingly relevant for CMOs and regulatory affairs teams managing rare cardiac disease submissions.

The approval was supported by Trial AG10-301, a two-arm, double-blind, parallel-design Phase 3 study enrolling 632 randomized patients across 95 sites in 18 countries. Subjects were randomized 2:1 to ATTRUBY 712 mg twice daily, equivalent to 800 mg acoramidis HCl, or matching placebo, and followed for 30 months. The trial used an embedded design: Part A assessed 12-month functional and health-related quality of life endpoints, while Part B evaluated 30-month mortality, morbidity, and functional endpoints. Part A did not meet its primary endpoint of change from baseline in 6-Minute Walk Distance at Month 12, with a difference in least-squares mean of -2 meters and a p-value of 0.76. Efficacy conclusions are drawn from Part B results. The safety population comprised 421 trial participants.

Regulatory and manufacturing context: For plant heads and QA directors, the 712 mg twice-daily dosing regimen and the distinction between free base and HCl salt forms (712 mg acoramidis free base equivalent to 800 mg acoramidis HCl) present formulation and content uniformity considerations that will require close attention during tech transfer and process validation under 21 CFR Part 211 and ICH Q10 frameworks. The multi-country, 95-site trial footprint also underscores the complexity of comparator supply chain management for rare disease studies.

Of the 632 randomized patients, 126 (20%) were from the United States, with the remainder enrolled across sites in the United Kingdom, Australia, Italy, Spain, Denmark, Canada, Czech Republic, New Zealand, Belgium, Israel, Netherlands, Greece, Brazil, Ireland, South Korea, Poland, and Portugal. Following the 30-month treatment period, all randomized subjects were eligible to enroll in the open-label extension trial AG10-304.

Source: FDA Drug Trials Snapshot for ATTRUBY, published April 29, 2026, based on original approval data from November 22, 2024. Full prescribing information should be consulted for approved conditions of use.