Acoramidis Shows Sustained Mortality Benefit in ATTR-CM Trial

Event summary

  • Long-term data from the ATTRibute-CM trial demonstrated a 44.7% reduction in all-cause mortality (ACM) and a 49.3% reduction in cardiovascular mortality (CVM) through Month 54 (p<0.0001).
  • Acoramidis mitigated the rise in NT-proBNP through Month 54 to a greater extent than observed with previous disease-modifying treatments.
  • Treatment with acoramidis stabilized and maintained heart failure-related quality of life scores (KCCQ-OS) through Month 54.
  • The findings were presented at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo and simultaneously published in JAMA Cardiology.

The big picture

ATTR-CM represents a significant unmet medical need, and while several therapies have emerged, patient outcomes remain suboptimal. BridgeBio’s acoramidis data suggest a meaningful improvement in survival and quality of life, but the real-world survey data highlights a gap between available treatment and patient access. The long-term data strengthens the company’s position in a competitive market, but sustained adoption will require addressing physician and patient concerns.

What we're watching

Commercialization
The real-world survey data highlighting physician dissatisfaction and patient undertreatment suggests challenges in acoramidis adoption despite efficacy, requiring BridgeBio to address awareness and access barriers.
Competitive Landscape
Given the emergence of other ATTR-CM therapies, the sustainability of acoramidis’ market share will depend on continued demonstration of superior long-term outcomes and a favorable cost-benefit profile.
Regulatory Scrutiny
Further data releases and post-market surveillance will be critical to maintaining regulatory approval and addressing any potential long-term safety concerns, particularly given the relatively high discontinuation rate due to adverse events.

Related topics

Drug Development