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Emerging Therapies

Advances in HAE Therapeutics Highlight Path Toward Personalized, Patient-Centered Care

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Ongoing innovations in hereditary angioedema (HAE) treatment are reshaping disease management, with novel therapies targeting key mechanisms in the kallikrein-kinin pathway and expanding options for both on-demand and prophylactic care, according to a recent narrative review.

HAE is a rare, autosomal dominant disorder caused by excessive bradykinin production, leading to recurrent episodes of subcutaneous and submucosal swelling. These unpredictable attacks can involve the gastrointestinal tract, extremities, oropharynx, or airway, posing significant morbidity and mortality risks. Historically, treatment has focused on controlling acute episodes, but recent therapeutic advances have introduced options for long-term prevention.

“Current on-demand therapies rapidly relieve acute symptoms, while contemporary prophylactic strategies have substantially reduced attack frequency and improved patient autonomy and health-related quality of life,” the authors reported.

The review outlines the evolution of HAE care, emphasizing the impact of precision therapies that disrupt specific elements of the bradykinin cascade. Advances include monoclonal antibodies, kallikrein inhibitors, and plasma-derived or recombinant C1 inhibitors, all of which have demonstrated efficacy in preventing or aborting attacks.

Emerging therapies aim to further simplify care. “Emerging therapies—including novel oral agents, monoclonal antibodies, RNA therapies, and pioneering gene editing approaches—continue to evolve,” the authors wrote. These next-generation options are designed to enhance convenience, durability, and tolerability while maintaining therapeutic efficacy.

The review emphasizes that innovation must be paired with equitable access and sustained treatment engagement.

These therapeutic advancements “collectively strive to address remaining unmet needs, ensuring broader accessibility, convenience, and long-term sustainability of care,” the authors concluded. The review reinforces the central role of targeted, patient-centered innovation in improving outcomes and quality of life for individuals living with HAE.

Reference
Uminski K, Goodyear D, Betschel S. Therapeutic Advances in Hereditary Angioedema: A Focus on Present and Future Options. Adv Ther. 2025;42(12):5879-5895. doi:10.1007/s12325-025-03382-6

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