NICE has cleared three HAE prophylaxis agents since 2019; two newer entrants remain under appraisal.
NICE has issued three positive technology appraisals for HAE prophylaxis in England: lanadelumab (Takhzyro, TA606, published 16 October 2019, recommended only for patients already eligible for C1-inhibitor prophylaxis under NHS England's commissioning policy), berotralstat (Orladeyo, TA738, published 20 October 2021, recommended for patients with two or more attacks per month), and garadacimab (Andembry, TA1101, published 8 October 2025, recommended on the same two-attacks-per-month threshold). Two newer MHRA-approved agents sit earlier in the pathway: donidalorsen (Dawnzera) has a NICE draft guidance consultation open, and sebetralstat (Ekterly) has draft guidance favourable to funding, but neither has a confirmed final NICE decision as of this brief.
The sequencing matters commercially: three years separate TA606 and TA1101, and each carries a confidential simple-discount patient access scheme, so list prices (berotralstat's annualised list of roughly £133,121 per NICE TA738; garadacimab's £20,625 per pre-filled pen per TA1101) understate real net cost. Sebetralstat bridges the pre-NICE gap via an MHRA Early Access to Medicines Scheme opinion from March 2025; donidalorsen has no confirmed interim NHS access route while its appraisal proceeds. Any UK market-access read must track appraisal status drug-by-drug, not assume a US or EU approval implies NHS funding.
Three NICE technology appraisals fund HAE prophylaxis in England; two newer agents remain in appraisal.
| Agent | Mechanism | UK Status | Key Evidence |
|---|---|---|---|
| Lanadelumab (Takhzyro, Takeda) | Anti-plasma kallikrein mAb, SC | NICE TA606 (16 Oct 2019): recommended, restricted to patients eligible for C1-inhibitor prophylaxis; simple discount PAS | HELP: 87% reduction in monthly attack rate vs placebo |
| Berotralstat (Orladeyo, BioCryst) | Oral plasma kallikrein inhibitor | NICE TA738 (20 Oct 2021): recommended for patients with ≥2 attacks/month | APeX-2: ~80% reduction in monthly attack rate vs placebo |
| Garadacimab (Andembry, CSL Behring) | Anti-Factor XIIa mAb, monthly SC | NICE TA1101 (8 Oct 2025): recommended for patients with ≥2 attacks/month | VANGUARD: 87% reduction in monthly attacks |
| Donidalorsen (Dawnzera, Ionis) | Antisense oligonucleotide, SC | MHRA-approved; NICE draft guidance consultation open, no confirmed final decision | OASIS-HAE: ~81% reduction in attack rate vs placebo |
| Sebetralstat (Ekterly, KalVista) | Oral plasma kallikrein inhibitor, on-demand | MHRA-approved Jul 2025, bridged by EAMS opinion Mar 2025; NICE draft guidance favourable, final pending | KONFIDENT: first oral on-demand HAE therapy |
Sources: NICE TA606, lanadelumab, published 16 October 2019; NICE TA738, berotralstat, published 20 October 2021; NICE TA1101, garadacimab, published 8 October 2025; NICE in-development project pages for donidalorsen and sebetralstat; NHS England Clinical Commissioning Policy for C1-esterase inhibitor prophylaxis (Jul 2016) and acute-attack treatment (2018); Longhurst et al., JACI In Practice, 2023; UK national audit of hereditary and acquired angioedema, Clin Exp Immunol, 2013.
What this brief answers
Every section answers a named commercial question your team is asking, scoped to your asset.
Delivers
- A drug-by-drug NICE appraisal status map distinguishing final technology appraisals from open consultations
Delivers
- The exact NICE eligibility criteria per technology appraisal, usable for funded-population sizing
Delivers
- UK-specific epidemiology and diagnostic-delay benchmarks to size the funded-versus-undiagnosed population gap
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Commission this briefWhat's inside
- TA606 lanadelumab, TA738 berotralstat, TA1101 garadacimab
- Restriction criteria per appraisal
- MHRA licensing status vs confirmed NICE funding
- EAMS bridge mechanism for sebetralstat
- C1-inhibitor and icatibant national commissioning policies
- The 37-centre national survey and 1,152-patient count
- Historical vs current diagnostic-delay benchmarks
- Why every NICE-recommended HAE agent carries a confidential simple-discount patient access scheme that understates its real net cost
- How list prices, berotralstat's roughly £133,121 annualised and garadacimab's £20,625 per pre-filled pen, compare against the discounted net price payers actually pay
- Why three years separate lanadelumab's TA606 and garadacimab's TA1101, and what that sequencing means for a new UK entrant
- How sebetralstat's EAMS bridge contrasts with donidalorsen's lack of any confirmed interim NHS access route
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How AXLRx builds this brief
Prepared by MoatRx analysts.
This brief sources findings directly from NICE's published technology appraisal guidance pages and NICE's in-development project pages for donidalorsen and sebetralstat, which show draft guidance issued but final guidance not yet confirmed as of this brief. UK epidemiology figures come from the peer-reviewed national survey of hereditary angioedema in the UK and the earlier UK national audit.
MHRA approval dates and regulatory status for donidalorsen and sebetralstat were cross-checked against regulatory press notices. NHS England commissioning policy for C1-esterase inhibitor prophylaxis and acute-attack treatment was sourced from the original NHS England policy documents. No US approval or reimbursement pattern was carried across to the UK by assumption.
- Every NICE technology appraisal number (TA606, TA738, TA1101) verified against the live NICE guidance page, not inferred from US FDA approval status
- Donidalorsen and sebetralstat UK status confirmed as in NICE appraisal with no final decision confirmed, based on live NICE in-development project pages
- UK prevalence and diagnostic-delay figures sourced from peer-reviewed UK national survey and audit publications, not extrapolated from US epidemiology
Frequently asked questions
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