Hereditary angioedema (HAE) is a rare, autosomal dominant genetic disorder characterized by recurrent, unpredictable, and potentially life-threatening attacks of subcutaneous and submucosal swelling, driven by uncontrolled bradykinin production and, in the most common forms, by deficiency or dysfunction of C1 esterase inhibitor (C1-INH). Over recent decades, pharmacotherapy for HAE has evolved from treatment of acute attacks towards reducing the risk of episodes occurring. The HAE market has matured into one of the most therapeutically crowded rare-disease landscapes, with management spanning on-demand (OD) treatment, short-term prophylaxis, and long-term prophylaxis (LTP), and plasma-derived C1-INH replacement increasingly displaced by more convenient subcutaneous and oral agents. The OD segment has been reshaped by KalVista's Ekterly, the first oral OD therapy, while the LTP market intensified sharply with two US launches in 2025, CSL's Andembry and Ionis's Dawnzera, as well as continued momentum for BioCryst's oral Orladeyo and Takeda's Takhzyro, making dosing convenience the principal driver of patient and prescriber choice. The pipeline is headlined by Intellia's lonvoguran ziclumeran (lonvo-z), an in vivo CRISPR gene-editing therapy that reported positive Phase III HAELO results in April 2026 and could launch as early as the first half of 2027, positioning it to disrupt the chronic-prophylaxis paradigm as the first one-time HAE treatment. Despite this innovation, HAE remains substantially underdiagnosed with diagnostic delays of years, and payer pressure and an increasingly fragmented competitive landscape remain significant barriers to translating therapeutic advances into real-world benefits.

This Datamonitor Healthcare report contains a Disease Analysis module.