NICE has cleared three HAE prophylaxis therapies since 2019, but donidalorsen and sebetralstat remain without a confirmed NHS funding decision.

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), 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 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 Two newer MHRA-approved agents sit earlier in the pathway: donidalorsen (Dawnzera) has a NICE draft guidance consultation open, and sebetralstat (Ekterly) has draft guidance favourable to funding, but neither has a confirmed final NICE decision as of this brief.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 and garadacimab's £20,625 per pre-filled pen, 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.

MHRA licensing does not mean NHS funding until NICE actually decides.

Five questions this report answers:

Q1 - Which HAE therapies does the NHS in England actually fund today versus only MHRA-license?

Q2 - What restriction criteria gate access within each NICE-recommended HAE prophylaxis agent?

Q3 - How do UK underdiagnosis and diagnostic delay affect the addressable treated HAE population?

Q4 - Which MHRA-licensed HAE agents remain without a confirmed NICE funding decision?

Q5 - How does sebetralstat's Early Access to Medicines Scheme bridge the pre-NICE funding gap?

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Mike || Global Pharma Commercial Marketing Head

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