Lanadelumab in Hereditary Angioedema - Real-World Outcomes and Implications for Access Practices in Europe

Q: What is lanadelumab used for in hereditary angioedema?

Lanadelumab is used for long-term HAE prevention. It is intended to reduce attacks and prevent bradykinin-mediated angioedema.

Q: What outcomes were observed after at least six months of lanadelumab treatment?

After 6 months, attacks dropped to a median of 0 and on-demand medication use decreased from 16 to 0 doses, with results persisting beyond 6 months.

Q: How did reimbursement criteria across Europe affect patient access?

Reimbursement models varied, with some using restrictions based on attack frequency or treatment response. The study suggests some patients who could benefit may be excluded under certain criteria.

Sep 30, 2026

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Lanadelumab is approved in the EU for long-term hereditary angioedema (HAE) prevention in patients aged ≥2 years. Despite strong trial efficacy, limited real-world evidence clarifies reimbursement effects and clinical outcomes. This retrospective study in Poland analyzed 72 patients receiving lanadelumab in the national drug program and compared European reimbursement policies, evaluating how effectiveness informs access restrictions. After 6 months, attacks fell to 0 and on-demand rescue use decreased to 0.

LanguageEnglish

CategoryHealthcare

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Pages13

Reading Time33 min

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Abstract

Introduction

Article Lanadelumab in Hereditary Angioedema: Real-World Outcomesand Implications for Access Practices in Europe Dagmara Róyk 1, Witold Wrona 2, *, Barbara Kucharczyk 3, Agata Tomaszewska 4,5 and Aleksandra Kucharczyk 6 Academic Editor: JürgenRech Received: 25 November 2025 Revised: 18 December 2025 Accepted: 22 December 2025 Published: 26 December 2025 Copyright: © 2025 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license. 1 Law Office Dagmara Róz˙ yk Attorney at Law, 00-127 Warsaw, Poland 2 HealthQuest Sp. z o.o., 01-517 Warsaw, Poland 3 Medical Faculty I, Medical University of Warsaw, 02-091 Warszawa, Poland 4 Department of Paediatrics, Nephrology and Allergology, Military Institute of Medicine-National Research Institute, 04-141 Warsaw, Poland 5 Faculty of Medicine, University of Warsaw, 00-927 Warsaw, Poland 6 Department of Internal Diseases, Pneumonology, Allergology, Clinical Immunology and Rare Diseases, Military Institute of Medicine-National Research Institute, 04-141 Warsaw, Poland * [Correspondence: witold.wrona@healthquest.pl](Correspondence: witold.wrona@healthquest.pl) Abstract Background/Objectives: Lanadelumab is approved in the EU for long-term HAE prevention in patients aged ≥2 years. While trials show high efficacy, real-world data on reimbursement and outcomes are limited. This study presents real-world clinical results in Poland and compares reimbursement criteria across European countries, assessing how effectiveness influences access restrictions. Methods: This retrospective analysis examined patients in the Polish drug program for lanadelumab. It collected demographics, disease features, attack frequency, and rescue medication use before and after at least six months of treatment. Additionally, a review of European reimbursement policies was conducted using health technology assessments, policy documents, and literature. Results: The data of 72 patients with HAE with C1 inhibitor deficiency were analyzed. The median followup was 20.0 months (IQR 15.0–25.0) . The median baseline attack frequency was 15 over 6 months. After 6 months of lanadelumab, attacks dropped to 0 (IQR 0.0–0.0; p < 0.001), with 77.8% achieving >90% reduction. Most remained attack-free beyond 6 months; on-demand medication use decreased from 16 to 0 doses (p < 0.001) . Outcomes persisted beyond 6 months. No demographic or baseline variables predicted response. No discontinuations due to adverse events. Reimbursement criteria across Europe vary, from broad access to restrictions based on attack frequency or treatment response, with differences in care settings. Conclusions: Data from Poland confirm lanadelumab nearly eliminates severe HAE attacks in practice, regardless of attack frequency. Some European reimbursement models may exclude patients who could benefit. Using real-world effectiveness evidence in policies could improve access and outcomes for HAE patients. Keywords: hereditary angioedema; lanadelumab; real-world evidence; reimbursement 1. Introduction Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of painful, disabling, and even life-threatening subcutaneous or submucosal edema. Attacks are unpredictable in frequency and severity, imposing significant physical and psychosocial burdens on patients’ daily lives [1] . All patients require access to ondemand therapy, and short-term prophylaxis may be necessary in specific situations (e.g., medical or dental procedures) . Long-term prophylaxis (LTP) is a crucial component of HAE management, recommended not only for patients with frequent or severe attacks but also in cases where attacks are unpredictable, associated with high psychosocial burden, or involving potentially life-threatening locations such as the larynx. Until recently, LTP options were limited mainly t

FAQs

What is lanadelumab used for in hereditary angioedema?

Lanadelumab is used for long-term HAE prevention. It is intended to reduce attacks and prevent bradykinin-mediated angioedema.

What outcomes were observed after at least six months of lanadelumab treatment?

After 6 months, attacks dropped to a median of 0 and on-demand medication use decreased from 16 to 0 doses, with results persisting beyond 6 months.

How did reimbursement criteria across Europe affect patient access?

Reimbursement models varied, with some using restrictions based on attack frequency or treatment response. The study suggests some patients who could benefit may be excluded under certain criteria.

Sep 30, 2026

Upload by Blue Pony