BridgeBio received FDA approval for Attruby (acoramidis), a near complete TTR stabilizer (≥90%), approved to reduce cardiovascular death and cardiovascular-related hospitalization in ATTR-CM patients. The FDA approval is based on positive results seen in the ATTRibute-CM Phase III study, where Attruby significantly reduced death, cardiovascular-related hospitalization, and improved quality of life.
Attruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. 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. Through near-complete TTR stabilization, Attruby has been shown to preserve the native function of TTR as a transport protein of thyroxine and vitamin A and to demonstrate benefit on cardiovascular outcomes.
The ATTRibute-CM Phase III study enrolled 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR. Participants were randomized 2:1 to receive Attruby or placebo for 30 months. As published in The New England Journal of Medicine, the trial successfully met its primary endpoint of a four-component composite endpoint of ACM, CVH, N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and six-minute walk distance with a Win Ratio of 1.8 (p<0.0001). Attruby demonstrated a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and six-minute walk test. Additionally, the increase in NT-proBNP on treatment was about half that of placebo.
Attruby was generally well-tolerated. The most common side effects were mild and included diarrhea and abdominal pain that were resolved without drug discontinuation. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively).
In as few as three months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalizations (CVH)) durably separated relative to placebo. A 42% reduction in composite ACM and recurrent CVH events relative to placebo at month 30. A 50% reduction in the cumulative frequency of CVH events relative to placebo at month 30.
“Transthyretin cardiac amyloidosis is a progressive disease with a poor prognosis when left untreated. Having a new first-line treatment option that provides excellent TTR stabilization and improves outcomes in this disease gives patients more options,” said Martha Grogan, MD, of the Mayo Clinic. “Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalization 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.”
The price is expected to be $18,759.12 for a 28-day supply. To honor the clinical trial participants, BridgeBio (Nasdaq: BBIO)—which was founded in 2015—will provide these patients Attruby free for life.
“With the landmark approval of Attruby, we gain the ability to serve patients with ATTR-CM. I’m grateful to each trial participant, their families, and the physicians, scientists, and our team at BridgeBio who made this possible,” said Neil Kumar, PhD, founder and CEO of Palo Alto-based BridgeBio. “Our journey is not over as we look to pursue approvals globally, next in Europe, Japan, and Brazil, and to continue exploring the full potential of this treatment. I am thrilled to extend our mission of ‘putting patients first’ with this third FDA approval in less than 10 years.”
BridgeBio submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe.
“We are excited to be part of the celebration for the FDA approval of Attruby. The need for more treatment options for patients living with ATTR-CM is crucial to achieving the goal of better outcomes and improved quality of life,” said Muriel Finkel, president of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers. “Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis.”
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