500,000+ US patients over 70 with HFpEF carry undiagnosed ATTRwt-CM. Two further sub-populations, Val122Ile carriers and ATTR-PN, remain even less visible.
The US ATTR-CM funnel is diagnosis-limited, not treatment-limited. An estimated 500,000+ patients aged 70 and older with heart failure with preserved ejection fraction (HFpEF) carry undiagnosed wild-type ATTR-CM (ATTRwt-CM), against only 70,000-100,000 currently diagnosed and treated. Annual new diagnoses run at 10,000-15,000 as Tc-PYP scintigraphy awareness grows among cardiologists, but that volume converts only a small share of the undiagnosed pool each year. One figure in the source research that looks like a contradiction is not: where a record cites a roughly 100,000-150,000 total ATTR-CM population, that number describes the diagnosed-and-modeled subset used for near-term commercial planning, not true prevalence. The 500,000+ estimate is the broader undiagnosed reservoir behind it, and this funnel carries both figures side by side rather than treating them as competing counts.
Two further sub-populations sit beside ATTRwt-CM, each with its own funnel. Hereditary ATTRv carriers, concentrated in the Val122Ile variant common among African Americans, number 100,000+ at an estimated 3-4% carrier rate, and most remain undiagnosed even though genetic testing is available. ATTR-PN (polyneuropathy) affects an estimated 5,000-10,000 patients, but a 4-5 year misdiagnosis delay, commonly mistaken for CIDP or diabetic neuropathy, leaves only 3,000-4,000 currently diagnosed and treated. All three funnels share the same rate-limiting step: cardiologists and neurologists ordering the confirmatory test, Tc-PYP scintigraphy for ATTR-CM and TTR genetic testing for ATTRv and ATTR-PN, rather than drug access or price.
US ATTR amyloidosis funnel — from undiagnosed reservoir to the treatment-eligible pool, by sub-population
| Funnel Stage | Population | Source |
|---|---|---|
| Undiagnosed ATTRwt-CM (HFpEF, age 70+) | 500,000+ | AXLRx ATTR launch-readiness research base (US) |
| Diagnosed and treated ATTRwt-CM today | 70,000-100,000 | AXLRx ATTR launch-readiness research base (US) |
| Annual new ATTR-CM diagnoses | 10,000-15,000/yr | AXLRx ATTR launch-readiness research base (US) |
| ATTRv (Val122Ile) carriers, mostly undiagnosed | 100,000+ (3-4% carrier rate) | AXLRx ATTR launch-readiness research base (US) |
| ATTR-PN estimated pool vs diagnosed and treated | 5,000-10,000 vs 3,000-4,000 | AXLRx ATTR launch-readiness research base (US) |
Sources: AXLRx ATTR amyloidosis disease-landscape, competitive-intelligence, payer-HTA, and launch-readiness research base (US), synthesized for this funnel.
What this model answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- The diagnosed-and-modeled subset (100,000-150,000) reconciled against the true undiagnosed reservoir (500,000+)
- Why both figures belong in the same funnel rather than in competition
Delivers
- The Val122Ile carrier pool (100,000+, 3-4% carrier rate), mostly undiagnosed
- The ATTR-PN misdiagnosis delay (4-5 years) and its 5,000-10,000 vs 3,000-4,000 diagnosed gap
Delivers
- 8-sheet structure (Strategic Context, Inputs, Model, Projections, Sensitivity, References, Market Context, QC)
- Live formulas, zero hardcoded cells; source citation per conversion step across all three sub-populations
Custom model delivered in 72 hours.
Commission This ModelWhat's inside
- Why diagnosis, not treatment access, sets the true addressable pool
- Reconciling the 100,000-150,000 diagnosed-subset figure against the 500,000+ undiagnosed reservoir before the rest of the model is built out
- 500,000+ US patients aged 70+ with HFpEF carrying undiagnosed ATTRwt-CM
- 100,000+ Val122Ile ATTRv carriers and 5,000-10,000 estimated ATTR-PN patients
- 70,000-100,000 diagnosed and treated ATTRwt-CM patients today
- 10,000-15,000 new diagnoses per year as Tc-PYP scintigraphy adoption grows
- How the three sub-populations diverge in diagnostic pathway and genetic testing rate
- 3,000-4,000 diagnosed ATTR-PN patients against a 5,000-10,000 estimated pool
- Part D routing for oral stabilizers vs Part B for infused or injected silencers
- The IRA orphan-drug exclusion and its effect on eligibility, not price
- Which assumptions move the eligible pool most across all three sub-populations
- Scenario ranges for undiagnosed-to-diagnosed conversion
- Patient volume by horizon under conservative, base, and aggressive scenarios
- Revenue translation inputs by sub-population
- The open questions your forecasting team must close before the model is finalised
- Structured for an internal forecast-review session
Included with every brief
How AXLRx builds this model
Prepared by MoatRx analysts.
Every AXLRx patient flow model is built on a five-layer funnel: population, disease burden (E1), diagnosis and specialist capture (E2), treatment and biomarker eligibility (E3), market access (E4), then Year 1-3-5 projections across three scenarios. Delivered as a live Excel workbook, not a static table, across 8 sheets with zero hardcoded cells.
US ATTR amyloidosis sources: AXLRx's own disease-landscape, competitive-intelligence, payer-HTA, and launch-readiness research base for the US market, reconciling the diagnosed-subset and true-prevalence figures into a single funnel.
- Undiagnosed ATTRwt-CM reservoir and diagnosed-and-treated population verified against the AXLRx US launch-readiness research base
- Val122Ile ATTRv carrier estimate and carrier rate verified against the AXLRx US launch-readiness research base
- ATTR-PN estimated pool and diagnosed-and-treated population verified against the AXLRx US launch-readiness research base
- Diagnosed-subset vs true-prevalence reconciliation checked against the AXLRx US disease-landscape and competitive-intelligence records
Frequently asked questions
Commission this model
AXLRx delivers rare-disease patient flow models built for forecasting and launch teams sizing the US ATTR amyloidosis opportunity across ATTRwt-CM, ATTRv, and ATTR-PN. Custom model in 72 hours.
Specify your indication, market, and cohort definition.
AXLRx analyst confirms funnel scope and comparator set before building.
Research-verified patient flow model in 72 hours with optional analyst readout.