Where ATTR cardiomyopathy treatment is heading

Transthyretin amyloid cardiomyopathy (ATTR-CM) is a stiffening of the heart caused by misfolded transthyretin protein, mostly in older men; it is now diagnosed in tens of thousands of Americans a year as imaging improves, and many more remain undiagnosed. Once fatal within a few years, it now has several drugs: stabilizers (tafamidis, acoramidis) and gene silencers (vutrisiran). The next wave aims to remove amyloid already in the heart, edit the gene once, or prevent disease in carriers.

• 77studies open or running
• 14phase 3 trials
• 449papers published this year

1. Now

   Nov 2026

   Gene silencing lowers deathsHappened 0.65 hazard ratio for death

   Vutrisiran cut death from any cause by 35% through 42 months. FDA approved March 2025.

   0.65: hazard ratio for death from any cause through 42 months.

   • HELIOS-B trial

   A second stabilizerHappened 35.9% death or heart hospitalization

   Acoramidis: death or heart hospitalization 35.9% vs 50.5%.

   35.9%: died or were hospitalized for heart causes, vs 50.5% with placebo.

   • ATTRibute-CM trial

   Not every silencer adds benefitHappened

   Eplontersen missed its main goal; people already on stabilizers saw no extra benefit.

   • CARDIO-TTRansform trial

2. 6 months

   by May 2027

   Choosing stabilizer, silencer or bothLikely 0.65 hazard ratio (vutrisiran)

   Guidelines will weigh HELIOS-B and CARDIO-TTRansform on whether to combine drug types.

   0.65: hazard ratio for death from any cause through 42 months.

   • HELIOS-B trial
   • CARDIO-TTRansform trial

3. 12 months

   by Nov 2027

   Removing amyloid already in the heartPossible

   Alexion's DepleTTR-CM (1,182 people) tests ALXN2220, an antibody that clears amyloid deposits; due December 2027.

   • DepleTTR-CM

   One-time gene editingPossible

   Intellia's MAGNITUDE (1,200 people) resumed after the clinical hold was lifted; due December 2027.

   • Nex-z (MAGNITUDE) clinical hold lifted
   • MAGNITUDE

4. 3 years

   by late 2029

   Another amyloid-removing antibodyPossible

   Novo Nordisk's CLEOPATTRA (1,280 people) is due June 2029.

   • CLEOPATTRA

   A twice-yearly silencerPossible

   Alnylam's nucresiran TRITON-CM (1,750 people) is due May 2030.

   • TRITON-CM

5. 5 years

   by late 2031

   Preventing disease in gene carriersLong shot 35.9% events (ATTRibute-CM)

   ACT-EARLY (587 people) tests whether acoramidis prevents disease in people carrying TTR mutations; due October 2031.

   35.9%: died or were hospitalized for heart causes, vs 50.5% with placebo.

   • ATTRibute-CM trial
   • ACT-EARLY

The research paths

Borrowed from other conditions

Open to join now

• ATTR cardiomyopathy

  NNC6019-0001 (amyloid-removing antibody)

  Phase 3 · 299 sites

  Locations and eligibility →

• ATTR cardiomyopathy

  Nucresiran

  Phase 3 · 266 sites

  Locations and eligibility →

• ATTR cardiomyopathy

  Nex-z gene editing

  Phase 3 · 138 sites

  Locations and eligibility →

• TTR gene carriers

  Acoramidis prevention

  Phase 3 · 104 sites

  Locations and eligibility →

See every recruiting ATTR-CM trial →

When the trials finish

Completion dates the trials registered. Results usually follow 3 to 12 months later.

What just happened

Standard treatment today

• Diagnosed, mild to moderate symptomsTafamidis or acoramidis (stabilizers), or vutrisiran (silencer).
• Hereditary ATTR with nerve diseaseA silencer (vutrisiran, patisiran, eplontersen) is preferred.
• Advanced heart failureDiuretics and heart-failure care; heart transplant for selected people.
• Gene carrier, no diseaseMonitoring; prevention trials under way.

See ATTR-CM trials recruiting now →