ATTR-CM Market Intelligence Update

Headline News

Tafamidis Dominance (Published Feb 9, 2026)

• 80% of new ATTR-CM cases now initiated on tafamidis (Vyndamax/Vyndaqel)
• Source: Multicenter analysis published February 9, 2026

Improved Survival Outcomes

• Patients diagnosed 2018-2021: Lived >5 years on average
• Tafamidis efficacy: Reduces all-cause death risk by ~29%
• Caveat: Protective effect more pronounced in younger patients and earlier-stage disease
• Implication: Early diagnosis is critical for optimal outcomes

Competitive Landscape

Three Recognized Therapies

All three treatments show substantial clinical benefit vs. no disease-specific therapy:

1. Tafamidis (Vyndamax/Vyndaqel, Pfizer)

- Market leader: 80% initiation rate

- Patent status: EU orphan exclusivity until February 2030

1. Acoramidis (Attruby, BridgeBio Pharma)

- Recognized as effective alternative

1. Vutrisiran (Amvuttra, Alnylam Pharmaceuticals)

- Recognized as effective alternative

Pricing & Access Concerns

Cost-Effectiveness Pressure

• Independent appraisal committee: Tafamidis pricing "much too high"
• Suggested cost-effective range: $13,600-$39,000 annually
• Strategic implication: Pricing pressure may drive:

- Shift to alternative therapies

- Generic entry post-2030 (EU)

- Increased scrutiny on treatment initiation and patient selection

Monitoring Challenges

No Direct Efficacy Test

Tafamidis effectiveness must be monitored indirectly through:

• Cardiac biomarkers (NT-proBNP, troponin)
• Echocardiography measurements
• Cardiac MRI imaging
• Functional tests (6-minute walk, KCCQ scores)
• Symptom assessments

Strategic Implications for Ada

Market Tailwinds

1. High initiation rates: 80% of new cases → large addressable market for patient identification
2. Early treatment imperative: Younger, earlier-stage patients benefit most → premium on early diagnosis
3. Monitoring gap: No simple efficacy test → opportunity for longitudinal patient tracking/outcomes data

Market Headwinds

1. Pricing pressure: May limit payer willingness to fund broad screening programs
2. Competitive alternatives: Three recognized therapies → treatment choice complexity
3. Patent expiration (2030 EU): Generic entry could commoditize TTR stabilizers, shift value to diagnosis/monitoring

Positioning Recommendations

1. Emphasize early diagnosis ROI: Show cost savings from treating earlier-stage disease vs. late-stage
2. Real-world evidence: Partner with academic centers to validate patient identification methods and treatment outcomes
3. Payer alignment: Frame patient finder as cost-effective given pricing pressure and need for appropriate patient selection
4. Monitoring solutions: Consider expanding beyond identification into treatment response tracking