BridgeBio Pharma has received FDA approval for Attruby (acoramidis), a novel treatment for adult patients with transthyretin amyloid cardiomyopathy (ATTR-CM). This approval marks a significant milestone, providing a new therapeutic option to reduce cardiovascular death and cardiovascular-related hospitalization in this patient population. Attruby is an orally administered, near-complete stabilizer of transthyretin (TTR).
The approval is based on the Phase 3 ATTRibute-CM study, which enrolled 632 participants with symptomatic ATTR-CM associated with either wild-type or variant TTR. The study met its primary endpoint, demonstrating a statistically significant treatment effect at 30 months with a Win Ratio of 1.8. The primary endpoint was a four-component composite including all-cause mortality (ACM), cardiovascular hospitalization (CVH), N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and six-minute walk distance.
Mechanism of Action
Attruby works by mimicking a naturally occurring 'rescue mutation' of the TTR gene, targeting the root cause of ATTR-CM: the destabilization of the native TTR tetramer. By stabilizing the TTR protein, Attruby prevents the formation of amyloid fibrils that deposit in the heart, leading to cardiac dysfunction.
Clinical Trial Results
The Phase 3 ATTRibute-CM study demonstrated that Attruby significantly reduced cardiovascular death and hospitalization, and improved quality of life. The drug was generally well-tolerated, with the most common side effects being mild, including diarrhea and abdominal pain.
According to Mayo Clinic MD Martha Grogan, "Transthyretin cardiac amyloidosis is a progressive disease with a poor prognosis when left untreated. Having a new first-line treatment option which provides excellent TTR stabilisation and improves outcomes in this disease gives patients more options. Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalisation as early as three months after initiation of therapy. With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition."
Patient Support
BridgeBio is offering a patient support services program called ForgingBridges to assist US patients and their families in accessing treatment.
Future Plans
BridgeBio plans to pursue approvals globally, next in Europe, Japan, and Brazil, to extend the availability of Attruby to more patients worldwide. This approval marks BridgeBio's third FDA approval in less than 10 years.
