FDA Approves Attruby for Heart Failure Indication

The FDA has approved BridgeBio’s Attruby (acoramidis), an orally administered therapy to treat adults with transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular death and cardiovascular-related hospitalization.

ATTR-CM is a type of heart failure. It is a rare disease that leads to a build-up of the protein transthyretin in the left ventricle of the heart, leading to a thickening of the heart muscle.

“Access to this new therapy means more hope and more opportunity to improve the lives of patients with amyloidosis,” Muriel Finkel, president of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers, said in a news release.

Attruby is an oral small molecule stabilizer of transthyretin. It was designed to mimic the protective effects of T119M mutation, which has been associated with reduced risk of vascular events. It will have a list price of $18,759 for a month’s supply.

BridgeBio offers a patient support services program, ForgingBridges, which includes insurance resources and financial assistance options. Commercial patients may be eligible for a $0 copay; Medicare patients will have a max copay of $167 per months, according to company officials.

The FDA approval is based on positive results seen in the ATTRibute-CM phase 3 study, which enrolled 632 patients with symptomatic ATTR-CM, and patient were randomly assigned to receive either Attruby or placebo. The trial met its primary endpoint of a four-component composite endpoint of ACM, including CVH, N-terminal prohormone of brain natriuretic peptide (NT-proBNP), and six-minute walk distance with a Win Ratio of 1.8. The win ratio is a method for reporting composite endpoints that gives priority to certain events. Patients in these trials are matched based on risk profiles and events are compared among the pairs and assigned a “win” if they meet the endpoint.

The study found that Attruby demonstrated a statistically significant treatment effect at 30 months on the Kansas City Cardiomyopathy Questionnaire and 6-minute walk test. Additionally, the increase in NT-proBNP on treatment was about half that of placebo.

BridgeBio also recently released data from an open-label extension acoramidis in ATTR-CM at the American Heart Association (AHA) Scientific Sessions and published in Circulation.

The open-label extension includes 389 participants who completed the 30-month. ATTRibute-CM phase 3 study confirmed results from the earlier study and found reduction of composite all-cause mortality and cardiovascular-related hospitalizations by 46% at month 36 and 48% at month 42. The open label study also found evidence of early benefit in patients who crossed over from placebo to acoramidis after month 30, compared with extrapolated placebo curve.