Last week, the FDA approved BridgeBio’s Attruby (acoramidis) for adults with Transthyretin amyloid cardiomyopathy (ATTR-C)—a rare disease that can cause serious heart problems and death. Attruby is the first and only approved treatment with a label specifying near-complete stabilization of Transthyretin (TTR). The company also said Attruby demonstrated the most rapid benefit seen in any Phase III study of ATTR-CM to date.
“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 stabilization and improves outcomes in this disease gives patients more options,” said Martha Grogan, MD, consultant in the Department of Cardiovascular Diseases, and director of the Cardiac Amyloid Clinic 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.”
Attruby was designed to mimic a naturally occurring “rescue mutation” of the TTR gene (T119M) that targets the root cause of ATTR-CM, destabilization of the native TTR tetramer. Through near-complete TTR stabilization, Attruby works by preserving the native function of TTR as a transport protein of thyroxine and vitamin A.
The FDA approval is based on positive results from the ATTRibute-CM Phase III study, where Attruby significantly reduced death and cardiovascular-related hospitalization, and improved quality of life. BridgeBio also submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025.
“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. Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis,” said Muriel Finkel, president of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers.
The ATTRibute-CM Phase III study enrolled 632 patients with symptomatic ATTR-CM, associated with either wild-type or variant TTR. Subjects were randomized 2:1 to receive Attruby or placebo for 30 months.
As recently reported in The New England Journal of Medicine, the trial successfully met its primary endpoint and six-minute walk distance test. 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 N-terminal pro b-type natriuretic peptide (NT-proBNP) was only about half on Attruby as that of placebo.
“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. “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 offers a patient support services program, ForgingBridges, for people in the U.S. prescribed Attruby and their families to receive help accessing the therapy.
