BridgeBio Pharma, Inc. announced the FDA approval of Attruby™ (acoramidis), an oral stabilizer of transthyretin (TTR), for adults with transthyretin amyloid cardiomyopathy (ATTR-CM). This treatment aims to reduce cardiovascular death and hospitalization, based on positive results from the ATTRibute-CM Phase 3 trial.
Key Highlights:
- Efficacy: Attruby demonstrated significant improvements in cardiovascular outcomes, quality of life, and functional capacity, with benefits observed as early as three months into treatment.
- Mechanism: It provides near-complete stabilization of TTR (≥90%), mimicking a protective TTR gene mutation (T119M) to preserve TTR’s natural functions.
- Clinical Data: In the Phase 3 study of 632 patients, Attruby significantly outperformed placebo on primary and secondary endpoints, including mortality reduction and improved walk test results.
Expert and Patient Support:
- Expert Insight: Dr. Martha Grogan of the Mayo Clinic highlighted that Attruby offers a much-needed first-line option for managing ATTR-CM as a chronic condition.
- Support Services: BridgeBio launched the ForgingBridges™ program to assist patients with access, insurance, and financial aid.
Global Expansion:
BridgeBio seeks global approval, with applications under review in Europe, Japan, and Brazil. Bayer will commercialize the drug in Europe.
Safety Information:
Common side effects include mild diarrhea and abdominal pain, with no significant differences in discontinuation rates between Attruby and placebo groups. Some reversible kidney function changes were noted.
