Acoramidis Snags FDA Approval for ATTR Cardiomyopathy

Acoramidis (Attruby) gained FDA approval for treating wild-type or variant transthyretin (TTR) amyloidosis cardiomyopathy (ATTR-CM), BridgeBio Pharma announced.

Offering patients near-complete TTR stabilization in an oral tablet, acoramidis is indicated to reduce cardiovascular death and cardiovascular-related hospitalization in adults with ATTR-CM (also known as cardiac ATTR amyloidosis), a rare but increasingly recognized cause of heart failure in which amyloid builds up in the heart due to a gene mutation.

“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, of the Mayo Clinic in Rochester, Minnesota, in a company press release.

Another oral TTR stabilizer, Tafamidis (Vyndamax, Vyndaqel), had held the title of the sole drug specifically indicated for the disease since 2019. Last year, the FDA declined to give the gene silencer patisiran (Onpattro) an indication for ATTR-CM despite a net-favorable FDA advisory committee vote just a month prior.

FDA approval for acoramidis was based on the ATTRibute-CM trial, in which the drug met the 30-month primary endpoint compared with placebo when pooling the outcomes of all-cause mortality, cumulative cardiovascular hospitalization frequency, change from baseline in N-terminal pro-brain natriuretic peptide (NT-proBNP), and change from baseline in 6-minute walk distance (6MWD).

That 632-person study could not show significant reduction in the individual endpoints of 30-day all-cause mortality or 6MWD at 12 months with acoramidis, as ATTRibute-CM might have been underpowered for those analyses. Notably, the trial protocol also allowed drop-in tafamidis use after month 12 at the discretion of the treating clinician, which may have diluted acoramidis’ benefits.

“Encouraging data suggests Attruby reduces all-cause mortality and cardiovascular hospitalization as early as 3 months after initiation of therapy,” Grogan said in her statement. “With continued advances in therapy, this previously fatal disease is becoming a manageable chronic cardiovascular condition.”

Acoramidis comes in 356-mg tablets and has a recommended dosage of 712-mg orally twice daily, according to its label.

In ATTRibute-CM, acoramidis was associated with a favorable safety profile. Side effects included diarrhea and upper abdominal pain, but most of these cases were deemed mild and resolved without drug discontinuation. Compared with the placebo group, acoramidis users showed a small increase in serum creatinine (0.2 vs 0.0 mg/dL with placebo) and decrease in estimated glomerular filtration rate (-8.2 vs -0.7 mL/min/1.73 m²) at day 28, both reversible after treatment discontinuation.

BridgeBio reportedly priced acoramidis at just under $19,000 for a 28-day supply. The company has indicated that it will be offering patients help with insurance and financial assistance for the drug through its patient support program, while trial participants continue to receive the drug free for life.

The company also announced that it has submitted acoramidis for approval in Europe (the decision is expected in 2025) and is also eyeing more regulatory clearances in Japan and Brazil.

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