The HAE prophylaxis class is splitting by route: one oral agent against four injectables, plus 2025's first oral on-demand treatment.

HAE prophylaxis has moved from androgens and injectable C1-esterase inhibitor replacement to a differentiated class of targeted agents. Long-term prophylaxis now spans the subcutaneous anti-kallikrein antibody lanadelumab (Takhzyro), the once-daily oral kallikrein inhibitor berotralstat (Orladeyo), C1-inhibitor replacement (Haegarda and Cinryze), and two 2025 entrants, the anti-Factor XIIa antibody garadacimab (Andembry) and the antisense agent donidalorsen (Dawnzera). On-demand treatment gained its first oral option in July 2025, sebetralstat (Ekterly), alongside established injectables icatibant and ecallantide.

The commercial contest is framed by route and dosing. Injectable antibodies post the largest attack-rate reductions, roughly 87% for lanadelumab and garadacimab and 81% for donidalorsen versus placebo, while oral berotralstat trades a more modest 44% reduction for daily convenience. Against a US prevalence near 1 in 50,000 and a diagnosed population of 6,000 to 10,000, HAE remains one of the most expensive US drug categories, with prophylaxis routinely exceeding $300,000 per patient per year.

Patients now trade attack-rate magnitude for convenience across five distinct prophylaxis mechanisms.

Five questions this report answers:

Q1 - How do the long-term prophylaxis agents differentiate on attack-rate reduction, route and dosing interval?

Q2 - What does the first oral on-demand agent, sebetralstat, mean for the acute-treatment market?

Q3 - How are US payers managing HAE access, and what does the ICER cost-effectiveness record imply for pricing?

Q4 - Why does injectable prophylaxis post 81 to 87% attack reduction against berotralstat's more modest 44%?

Q5 - What annual per-patient cost places HAE among the most expensive US drug categories?

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Mike || Global Pharma Commercial Marketing Head

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