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Transthyretin Cardiac Amyloidosis

ATTR-CM is a progressive infiltrative heart disease caused by misfolded transthyretin protein, routinely mistaken for common heart failure with preserved ejection fraction and left undiagnosed for years.
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About Transthyretin Cardiac Amyloidosis

In numbers

  • Diagnosed ATTR-CM prevalence in the USA is forecast to rise from 60.9 per million in 2025 to 99.3 per million by 2030, around 16,700 confirmed cases growing to 28,300.
  • Model-based estimates put true USA prevalence at 150,000 to 500,000 people, meaning most cases sit outside the diagnosed pool.
  • ATTR-CM is found in 10.2 per cent of people diagnosed with heart failure with preserved ejection fraction, and in 24 per cent of high-risk HFpEF patients in European cohorts.

Special details

  • Wild-type ATTR-CM affects predominantly men over 65, with a male to female ratio of around 10 to 1 and 85 per cent of diagnosed cases aged 65 or older.
  • Hereditary ATTR-CM linked to the Val122Ile variant carries a carrier frequency of about 3.4 per cent in people of West African ancestry, representing roughly 1.5 million carriers in the USA.
  • Other hereditary variants cluster geographically, including Val30Met in Portugal, Sweden and Japan, and Thr60Ala in Irish ancestry populations.
Signs and symptoms of Transthyretin Cardiac Amyloidosis
Diagnosis typically takes 3 to 5 years from first cardiac presentation, with soft-tissue signs such as bilateral carpal tunnel syndrome often present 5 to 10 years before cardiac diagnosis is confirmed.
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Breathlessness on exertion

Breathlessness on exertion

Heart failure with preserved ejection fraction, causing dyspnoea, orthopnoea and swelling in the legs.

Bilateral carpal tunnel syndrome

Bilateral carpal tunnel syndrome

Often appears 5 to 10 years before cardiac diagnosis and is found in 7.1 per cent of carpal tunnel patients overall.

Low-voltage ECG with thickened heart walls

Low-voltage ECG with thickened heart walls

A discordance between electrical voltage and muscle mass that is a distinctive but frequently missed signal.

Conduction problems

Conduction problems

Atrioventricular block and irregular heart rhythms, including atrial fibrillation in around 40 per cent of patients at diagnosis.

Tendon and joint signs

Tendon and joint signs

Biceps tendon rupture and spinal stenosis, both more common in people who go on to receive an ATTR-CM diagnosis.

Key challenges from product development to launch

Strategy and Business

Strategy and Business

  • The wrong disease or indication selected for investment
  • Indications tackled in the wrong order due to shaky success estimates
  • R&D returns slipping below the cost of capital
Clinical Development

Clinical Development

  • Trials delayed by low patient recruitment
  • High screen-failure rates that raise trial cost
  • Mixed cohorts that make the signals difficult to detect
HEOR, Access & Value

HEOR, Access & Value

  • A value case that arrives too late or is underdeveloped
  • Eligible population under-counted, budget-impact case unsubstantiated
  • Approved by the regulator, delayed at HTA
Medical Affairs

Medical Affairs

  • Fragmented, differently-coded healthcare data
  • Diagnostic delay that is long and unequal
  • No shared evidence base across functions
Launch and Commercial

Launch and Commercial

  • Launch planned without a view of the number of findable patients
  • Targeting too broad a population slows early uptake
  • RWE started too late to build the case

How Volv Global Helps

The earliest pipeline decisions carry lasting consequences: the wrong indication chosen, indications pursued in the wrong order, or capital committed where expected returns already trail its cost. Mistakes made this early are expensive to correct later.

inTrigue

Finds the true reachable population per candidate, so indication choices rest on evidence.

volv.global/finding-patients

inFlow

Adds probability-of-success and outcome evidence to help prioritise the pipeline.

volv.global/predict-patient-outcomes

When patients cannot be found, recruitment stalls and the burn rate climbs. High screen-failure rates add further cost and delay, and a mixed cohort makes the results harder to interpret.

inTrigue

Finds the undiagnosed and mis-coded patients conventional feasibility misses, widening the recruitable pool.

volv.global/finding-patients

inCare

Surfaces diagnosed-but-hidden, trial-eligible patients already in the data, so sites enrol faster.

volv.global/stratifying-patient-cohorts

inFlow

Predicts who will progress or respond, enriching the cohort so the signal is not diluted.

volv.global/predict-patient-outcomes

Without strong, timely evidence, payers can restrict access or cut the price. An under-characterised population weakens the budget case, and HTA approval demands more evidence than regulatory approval alone.

inFlow

Generates the real-world evidence and outcomes payers ask for, ahead of the submission deadline.

volv.global/predict-patient-outcomes

inTrigue

Finds the undiagnosed patients missing from the count, so the eligible population holds up under scrutiny.

volv.global/finding-patients

inCare

Surfaces the diagnosed-but-eligible patients who expand the eligible population count.

volv.global/stratifying-patient-cohorts

Healthcare data is fragmented across markets, so evidence has to be rebuilt market by market, slowing decisions. Time to an accurate diagnosis varies by who the patient is and where they are treated.

inTrigue

Works across fragmented, differently-coded data by design, so insight holds up country to country without a rebuild.

volv.global/finding-patients

inAdvance

Detects patients ahead of typical diagnosis, closing the gap in awareness and pathway that drives unequal delay.

volv.global/earlier-detection

A launch planned without a view of findable patients is poorly resourced from day one. Targeting too broad a population slows early uptake, and real-world evidence gathered too late leaves little time to build a strong access dossier.

inTrigue

Finds the treatable patients, so the launch is planned against real demand.

volv.global/finding-patients

inCare

Adds the eligible, reachable patients that launch planning is missing.

volv.global/stratifying-patient-cohorts

inFlow

Builds the access evidence early enough to matter.

volv.global/predict-patient-outcomes

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