Tafamidis built the ATTR-CM franchise as the only NICE-recommended stabiliser (TA984) — now acoramidis is recommended head-to-head (TA1121), with NICE directing clinicians to the least-expensive stabiliser.
Transthyretin amyloidosis (ATTR) is two commercial markets in one disease. ATTR cardiomyopathy (ATTR-CM) is served by oral TTR-tetramer stabilisers: tafamidis (Vyndaqel/Vyndamax, Pfizer), recommended by NICE in TA984 on the ATTR-ACT trial, and acoramidis (Beyonttra in the EU/UK, licensed to Bayer; marketed as Attruby in the US by BridgeBio), recommended by NICE in TA1121 in January 2026 on ATTRibute-CM as the first direct stabiliser competitor. ATTR polyneuropathy (ATTR-PN) is held by Alnylam's RNA-based TTR silencers: the siRNAs patisiran (Onpattro, IV, recommended under NICE HST10) and vutrisiran (Amvuttra, SC, recommended under NICE TA868). Vutrisiran has since crossed into cardiomyopathy, recommended by NICE in TA1115, opening a stabiliser-versus-silencer contest inside ATTR-CM.
The UK commercial story is defined by NICE mechanics rather than US list-price and IRA dynamics. Every UK recommendation rests on a confidential commercial arrangement (patient access scheme) that sets the net NHS price below list; vutrisiran's list price alone is about £383,000 per patient per year. In TA1121, NICE went further: because acoramidis and tafamidis are clinically similar and treat the same population, NICE directs clinicians to use the least expensive of the two stabilisers, having accounted for administration costs, dose, price per dose and commercial arrangements — a NICE-mandated cost-minimisation that converts the two-stabiliser contest into direct price competition. Access also diverges across the UK: a positive NICE technology appraisal carries a legal NHS funding obligation in England within 90 days, whereas Scotland requires separate acceptance by the Scottish Medicines Consortium (tafamidis was accepted by the SMC in November 2023 under the end-of-life and orphan medicine process, after an earlier rejection on cost-effectiveness grounds).
NICE-recommended transthyretin amyloidosis therapies — United Kingdom, 2026
| Drug (Brand / INN) | Mechanism | Company | Indication | NICE Status | Key Trial Result |
|---|---|---|---|---|---|
| Vyndaqel / Vyndamax (tafamidis) | Oral TTR-tetramer stabiliser | Pfizer | ATTR-CM | NICE TA984 — recommended (commercial arrangement) | ATTR-ACT: all-cause mortality −29.5% at 30 months vs placebo |
| Beyonttra / Attruby (acoramidis) | Oral TTR-tetramer stabiliser | Bayer (EU/UK) / BridgeBio | ATTR-CM | NICE TA1121 — recommended Jan 2026 (use least-expensive stabiliser) | ATTRibute-CM: mortality + CV hospitalisation 64.5% vs 74.0% at 30 months |
| Amvuttra (vutrisiran) | siRNA TTR silencer (SC) | Alnylam | ATTR-PN; ATTR-CM | ATTR-PN: NICE TA868, recommended (2023); ATTR-CM: NICE TA1115, recommended | HELIOS-A: mNIS+7 −2.2 vs +14.8 external control |
| Onpattro (patisiran) | siRNA TTR silencer (IV) | Alnylam | ATTR-PN | NICE HST10 — recommended (simple discount PAS) | APOLLO: mNIS+7 −34.0 vs +24.9 placebo |
Sources: NICE guidance TA984 (tafamidis), TA1121 (acoramidis), TA868 and TA1115 (vutrisiran), HST10 (patisiran); ATTR-ACT, NEJM 2018 (PMID 30145929); APOLLO, NEJM 2018 (PMID 29972753); HELIOS-A, Amyloid 2022 (PMID 35875890); ATTRibute-CM per BridgeBio (NEJM 2024).
What this brief answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- • Stabiliser vs silencer mechanism and the ATTR-CM vs ATTR-PN indication split • Efficacy across ATTR-ACT, ATTRibute-CM, APOLLO and HELIOS-A • Oral vs IV vs SC administration and monitoring burden by agent • Where vutrisiran's TA1115 cardiomyopathy recommendation resets the ATTR-CM contest
Delivers
- • ATTRibute-CM composite result and the clinical-differentiation argument • NICE cost-minimisation directive and its effect on net-price competition • Commercial-arrangement (PAS) dynamics between two clinically similar stabilisers • Prescriber switching considerations in a chronic, high-cost oral market
Delivers
- • NICE technology appraisal route, mandatory funding obligation and 90-day adoption in England • Confidential patient access schemes and their effect on net NHS price • SMC Scotland divergence, PACE and end-of-life/orphan flexibilities • The National Amyloidosis Centre pathway and Tc-PYP diagnostic scale-up
Custom brief delivered in 72 hours.
Commission This BriefWhat's inside
- Why ATTR-CM is served by oral TTR-tetramer stabilisers tafamidis and acoramidis while ATTR-PN is held by Alnylam's RNA-based silencers
- How vutrisiran's crossover into cardiomyopathy under NICE TA1115 opens a stabiliser-versus-silencer contest inside ATTR-CM itself
- Full profiles of tafamidis (NICE TA984), acoramidis (NICE TA1121), and the siRNA silencers patisiran (HST10) and vutrisiran (TA868/TA1115)
- Mechanism, sponsor, indication and NICE status for all four agents, from Pfizer's ATTR-ACT trial through BridgeBio/Bayer's ATTRibute-CM
- Why NICE's TA1121 recommendation directs clinicians to use the least expensive of the two clinically similar stabilisers
- How this cost-minimisation directive converts the ATTR-CM stabiliser contest into direct net-price competition between the two sponsors
- Why vutrisiran's NICE TA1115 recommendation extends Alnylam's silencer franchise beyond ATTR-PN into the larger ATTR-CM market
- How patisiran's IV administration under HST10 contrasts with vutrisiran's subcutaneous route as Alnylam's newer ATTR-PN option
- Why every UK recommendation rests on a confidential patient access scheme setting net NHS price below vutrisiran's roughly £383,000 list price
- How England's 90-day mandatory NICE funding obligation diverges from Scotland's separate SMC acceptance process, seen in tafamidis's pathway
- What comes after the current four-agent NICE-recommended landscape spanning TTR-stabiliser and RNA-silencer mechanisms
- How the National Amyloidosis Centre pathway and Tc-PYP diagnostic scale-up shape which patients reach these NICE-recommended therapies
Included with every brief
How AXLRx builds this brief
Prepared by MoatRx analysts.
Every AXLRx brief is built from primary regulatory and HTA sources: MHRA and EMA authorisations, NICE technology appraisals, NHS England commissioning documentation and Scottish Medicines Consortium advice, together with peer-reviewed trial literature, not secondary summaries, market-research reports, or unverified estimates. Findings are independently verified before inclusion; if a figure cannot be sourced to a live record, it does not ship.
UK ATTR Amyloidosis Competitive Intelligence sources: NICE guidance (TA984, TA1121, TA868, TA1115, HST10), the primary trial publications in the New England Journal of Medicine and Amyloid (ATTR-ACT, APOLLO, HELIOS-A), MHRA product information, and Scottish Medicines Consortium advice for NHS Scotland.
- Tafamidis ATTR-CM mortality result verified against ATTR-ACT, NEJM 2018 (PMID 30145929)
- Patisiran and vutrisiran polyneuropathy results verified against APOLLO (PMID 29972753) and HELIOS-A (PMID 35875890)
- NICE recommendations verified against NICE guidance TA984, TA1121, TA868, TA1115 and HST10 (nice.org.uk)
- SMC Scotland acceptance of tafamidis verified against Scottish Medicines Consortium advice (SMC2585, November 2023)
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