NHS Tc-PYP scintigraphy lifted estimated annual UK ATTR-CM diagnoses to 2,000–5,000, up from fewer than 500 a year before the pathway existed, and NICE's TA696 tafamidis commissioning, updated by TA984 in June 2024, is scaling to match.
Transthyretin (ATTR) amyloidosis results from destabilisation of the TTR protein, causing amyloid fibril deposition in the heart (ATTR-CM) and peripheral nerves (ATTR-PN), in wild-type or hereditary (ATTRv) form. NHS England established the Tc-PYP scintigraphy diagnostic pathway for ATTR-CM in 2021; approximately 30 NHS scintigraphy centres are now active, with referral running from HFpEF with increased echo wall thickness through CMR (where available) to Tc-PYP confirmation at an ATTR specialist centre. Estimated UK ATTR-CM incidence has risen to 2,000–5,000 new diagnoses per year, up from fewer than 500 per year before Tc-PYP availability.
ATTRv (hereditary ATTR) affects an estimated 1,000–2,000 UK patients; NHS GMS offers free TTR gene panel testing for probands with clinical features, and the UK National ATTRv Registry (led by UCL and Queen Elizabeth Hospital Birmingham) tracks approximately 800 patients. Val30Met is the most common variant, concentrated in Portuguese- and Brazilian-origin families in the UK, alongside Irish Thr60Ala families. NICE TA696 tafamidis approval, updated by TA984 in June 2024, remains the step-change commissioning event; vutrisiran (TA868, published 15 February 2023) was recommended for ATTR-PN with a simple Patient Access Scheme discount.
UK ATTR treatment landscape — NICE-commissioned tafamidis (TA696/TA984) and vutrisiran's PAS-backed ATTR-PN recommendation (TA868)
| Drug | Class | Company | MHRA/NICE Status | Key Evidence |
|---|---|---|---|---|
| Vyndaqel (tafamidis) | Oral TTR stabiliser | Pfizer | NICE TA696 with PAS, updated by TA984 (2024); NHS commissioned — 25 specialist centres | ATTR-ACT trial; approved 2019; NICE TA696, updated by TA984 (June 2024) |
| Amvuttra (vutrisiran) | siRNA, subcutaneous | Alnylam | NICE TA868 with simple PAS discount — ATTR-PN specialist centres | HELIOS-A trial; NICE TA868 (15 February 2023) |
Sources: NHS England ATTR-CM service specification 2023; NICE TA696/TA984 (tafamidis) evidence submissions; NICE TA868 (vutrisiran) evidence submission; UK National ATTRv Registry 2023.
What this assessment answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- Pre/post-Tc-PYP diagnosis rate comparison
- 30-centre scintigraphy network mapping
- NICE TA696/TA984 commissioned population sizing
Delivers
- UK National ATTRv Registry cohort characterisation
- TTR gene panel testing pathway
- variant distribution (Val30Met, Thr60Ala) by family origin
Delivers
- ATTR-PN diagnostic tools (nerve conduction, biopsy) and cohort sizing
- TA868 PAS-discount access structure
- specialist centre network (UCL, Oxford, QE Birmingham, Edinburgh)
Custom assessment delivered in 72 hours.
Commission This AssessmentWhat's inside
- How TTR protein destabilisation causes amyloid fibril deposition in the heart (ATTR-CM) and peripheral nerves (ATTR-PN), in wild-type or hereditary form
- Why Val30Met, concentrated in Portuguese- and Brazilian-origin UK families, and Irish Thr60Ala are the most common hereditary ATTRv variants
- How NHS England's 2021 Tc-PYP scintigraphy pathway lifted estimated UK ATTR-CM diagnoses from under 500 to 2,000-5,000 a year
- The referral chain from HFpEF with increased echo wall thickness through CMR to Tc-PYP confirmation across roughly 30 active NHS centres
- How the UK National ATTRv Registry, led by UCL and Queen Elizabeth Hospital Birmingham, tracks roughly 800 of an estimated 1,000-2,000 UK ATTRv patients
- Why NHS GMS offers free TTR gene panel testing for probands with clinical features, feeding registry identification
- How ATTR-CM cardiac burden and ATTR-PN neurological burden differ across the wild-type versus hereditary patient populations
- Why the rapid rise in NHS-diagnosed ATTR-CM cases since 2021 reflects a previously underdiagnosed burden, not a true incidence increase
- Tafamidis's NICE TA696 (updated TA984, June 2024) commissioning across 25 specialist centres against vutrisiran's TA868 ATTR-PN recommendation
- How the ATTR-PN specialist centre network (UCL, Oxford, QE Birmingham, Edinburgh) delivers vutrisiran under its Patient Access Scheme discount
- What changed between NICE TA696's original tafamidis approval and its TA984 update in June 2024
- Why vutrisiran's TA868 recommendation (15 February 2023) for ATTR-PN carries a simple Patient Access Scheme discount
Included with every brief
How AXLRx builds this assessment
Prepared by MoatRx analysts.
UK ATTR disease landscape is built from NHS England's ATTR-CM service specification, NICE's TA696/TA984 tafamidis evidence submissions, the UK National ATTRv Registry, and the UK ATTR-PN specialist network data underpinning vutrisiran's TA868 recommendation. The Tc-PYP pathway rollout since 2021 makes recent UK diagnosis-rate data unusually current relative to most ATTR markets.
Key sources: NHS England ATTR-CM service specification 2023; NICE TA696 and TA984 evidence submissions (tafamidis); NHS GMS TTR gene panel specification; UK National ATTRv Registry 2023 (UCL, QE Birmingham); UK ATTR-PN specialist network data; NICE TA868 evidence submission (vutrisiran). All figures carry source citations and are triangulated across multiple primary sources.
- ATTR-CM annual diagnosis estimates verified against NHS England ATTR-CM service specification 2023 and NICE TA696/TA984 evidence submissions
- ATTRv registry cohort and variant distribution verified against UK National ATTRv Registry 2023 (UCL and QE Birmingham)
- ATTR-PN cohort sizing verified against UK ATTR-PN specialist network data and NICE TA868 evidence (vutrisiran)
- NHS Tc-PYP scintigraphy centre count verified against NHS England ATTR-CM service specification 2023
Frequently asked questions
Commission this assessment
AXLRx ATTR Amyloidosis Disease Landscape is built for commercial, medical affairs, and market access teams that need a rigorous, evidence-based characterisation of the UK ATTR-CM and ATTRv patient populations and the NHS access pathway. Custom assessment in 72 hours.
Specify indication, geography, and epidemiological focus.
AXLRx analyst confirms subpopulation scope, data sources, and delivery format.
Research-verified assessment in 72 hours with optional analyst readout.