FDA Approves BridgeBio Pharma's Drug For Rare Heart Disease, Poised To Challenge Pfizer In Lucrative Yet Competitive Market

On Friday, the FDA approved BridgeBio Pharma, Inc.’s BBIO Attruby (acoramidis), an orally-administered near-complete (≥90%) stabilizer of Transthyretin (TTR) for adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization.

The FDA approval is based on ATTRibute-CM Phase 3 study results, which showed that Attruby significantly reduced death and cardiovascular-related hospitalization and improved quality of life.

Also Read: BridgeBio Pharma’s Acoramidis Shows Competitive Edge Against Pfizer’s Tafamidisa In Lucrative But Competitive ATTR-CM Market

Attruby is the first and only approved product with a label specifying near-complete stabilization of TTR.

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 3 study enrolled 632 participants with symptomatic ATTR-CM associated with either wild-type or variant TTR.

BridgeBio submitted a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025.

BridgeBio has granted Bayer AG BAYRY exclusive rights to commercialize acoramidis for ATTR-CM in Europe.

The company said it will provide Attruby for free to patients who participated in the trial.

William Blair writes, “We continue to view a reduction in all-cause mortality on label as a necessary distinction to gain major market share in ATTR-CM alongside Pfizer Inc’s PFE tafamidis.”

Since the Attruby label doesn’t include data on overall mortality, the William Blair analyst notes Pfizer might shift its marketing focus to emphasize claims about mortality benefits.

Price Action: BBIO stock is up 24.3% at $29.10 during the premarket session at last check Monday.

Image via Shutterstock

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