Growing the never-prophylaxed HAE cohort beats switching stable lanadelumab patients, before donidalorsen's 69% attack-reduction data resets the oral bar.

Lanadelumab (Takhzyro) holds roughly 45% of US HAE prophylaxis share with high KOL loyalty and very low breakthrough-attack rates in stable patients, a population that is structurally hard to switch. Berotralstat (Orladeyo), the once-daily oral entrant, has instead grown the market by converting never-prophylaxed patients, reaching roughly 20% share since 2020. Of the 8,000-9,000 US HAE patients, only 35-40% currently receive any prophylaxis; an estimated 2,500-4,000 patients meet prophylaxis criteria, three or more attacks a year or a laryngeal history, but remain untreated. This gap, not the stable lanadelumab base, is where a new entrant should aim.

The competitive clock is running: donidalorsen, KalVista's oral plasma-kallikrein inhibitor, reported a 69% attack-rate reduction in ZENITH-1 versus berotralstat's 44% in APeX-2, with an FDA submission in 2024 and possible US approval by 2025. Any new pre-launch entrant now competes not just against the two approved agents but against a pipeline drug likely to reset the oral efficacy bar before launch. A distinct commercial niche, the 1,000-1,500 US patients with FXII-HAE or other normal-C1-INH variants who may respond less well to standard kallikrein-targeted prophylaxis, remains structurally underserved by all three.

A pipeline drug is likely to reset the oral efficacy bar before launch.

Five questions this report answers:

Q1 - Should a new HAE agent target never-prophylaxed patients or stable lanadelumab switches?

Q2 - How does donidalorsen's pipeline data change the oral efficacy bar and launch timing?

Q3 - What PA criteria and value benchmark should a new HAE entrant plan against?

Q4 - What deliverable formats come with a commissioned US HAE launch readiness assessment?

Q5 - Which patient population should a pre-launch HAE prophylaxis asset target in the US?

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Mike || Global Pharma Commercial Marketing Head

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