Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence
- PMID: 40769455
- DOI: 10.1016/j.anai.2025.07.025
Lanadelumab's impact on hereditary angioedema control and quality of life across disease activity subgroups: Real-world evidence
Abstract
Background: Real-world clinical data support effectiveness and safety of lanadelumab in patients with hereditary angioedema (HAE); however, disease activity between patients can vary substantially in the absence of long-term prophylactic treatment.
Objective: To assess the effectiveness of lanadelumab in patients with HAE by baseline HAE attack frequency.
Methods: Patients with HAE from the phase 4 EMPOWER (NCT03845400) and ENABLE (NCT04130191) studies with available baseline attack rate data were included in this post hoc analysis. Disease activity subgroups were defined per pre-enrollment/lanadelumab (baseline) HAE attack rate (low, <1; moderate, ≥1 to <2; high, ≥2 to <3; very high, ≥3 attacks/mo).
Results: The analysis included 152 patients (low disease activity, n = 29; moderate, n = 29; high, n = 15; very high, n = 79). In all 4 subgroups, mean and median HAE attack rates after lanadelumab initiation were low (0.0-0.5 attacks/mo). Clinically meaningful improvements (≥6-point decreases) in mean Angioedema Quality of Life total scores were observed regardless of pre-lanadelumab attack rates. From month 1 after lanadelumab initiation to the end of follow-up, mean Angioedema Control Test Scores were 10 or more (indicating patient perception of well-controlled disease) in all 4 subgroups.
Conclusion: In these real-world data sets, on-treatment lanadelumab attack rates were low regardless of baseline disease activity. Patients from all 4 subgroups experienced improvements in health-related quality of life and disease control. Overall, these findings support long-term prophylaxis with lanadelumab across disease activity levels.
Trial registration: EMPOWER: ClinicalTrials.gov Identifier: NCT03845400; ENABLE: ClinicalTrials.gov Identifier: NCT04130191.
Copyright © 2025 The Authors. Published by Elsevier Inc. All rights reserved.
Conflict of interest statement
Disclosures Dr Andrea Zanichelli reports receiving speaker/consultancy fees from Astria Therapeutics, BioCryst, CSL Behring, KalVista Pharmaceuticals, Pharming, Pharvaris, Otsuka, and Takeda. Dr Walter A. Wuillemin reports receiving research grant support and speaker/consultancy fees from BioCryst, CSL Behring, and Takeda. Dr Emel Aygören-Pürsün has received honoraria, research funding, travel grants, and/or consulting fees from Astria Therapeutics, BioCryst, CSL Behring, Intellia Therapeutics, KalVista Pharmaceuticals, Otsuka Pharmaceutical, Pharvaris, and/or Takeda. Dr Aleena Banerji reports receiving research funding from Astria Therapeutics, Intellia Therapeutics, and Ionis Pharmaceuticals; and participated in advisory boards for Astria Therapeutics, CSL Behring, Intellia Therapeutics, Ionis Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, and Takeda. Dr Paula J. Busse has received research support and served on advisory boards for ADARx, Astria Therapeutics, BioCryst, CSL Behring, Intellia Therapeutics, KalVista Pharmaceuticals, and Takeda; and is a consultant for CVS Pharmacy. Dr Stephen D. Betschel reports receiving speaker and/or advisor fees from Astria Therapeutics, BioCryst, CSL Behring, Ionis Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, and Takeda; and research funding from CSL Behring and Takeda. Dr Mauro Cancian reports receiving personal fees from BioCryst, CSL Behring, KalVista Pharmaceuticals, Pharvaris, and Takeda. Dr Remi Gagnon reports site research at ALK, AstraZeneca, DBV Technologies, GlaxoSmithKline, Moderna, Novartis, Regeneron, and Sanofi; consultancy for ALK, Bausch Lomb, Regeneron, and Sanofi-Genzyme; and speaker fees from Bausch Lomb and Novartis. Dr M. Dawn Goodyear reports advisory boards/speaker fees from BioCryst, CSL Behring, and Takeda. Dr Tamar Kinaciyan reports receiving research funding, speaker/consultancy fees, and travel grants from BioCryst, CSL Behring, KalVista Pharmaceuticals, Kiniksa Pharmaceuticals, Novartis, Pharvaris, Sanofi, and Takeda. Dr Aharon Kessel reports receiving travel grants from Pharming and Takeda and honoraria from CSL Behring and Takeda. Dr Markus Magerl reports receiving research grant support and/or speaker/consultancy fees from Astria Therapeutics, BioCryst, CSL Behring, Intellia Therapeutics, Ionis Pharmaceuticals/Otsuka, KalVista Pharmaceuticals, Octapharma, Pharvaris, and Takeda. Dr Andreas Recke reports receiving research grants from Deutsche Forschungsgemeinschaft and Euroimmun; other research support from CSL Behring, Novartis, Pharming, Stallergenes Greer, and Takeda; honoraria and/or travel grants from BENCARD, BioCryst, CSL Behring, EUROIMMUN, Novartis, and Takeda; and served as a consultant or participated in advisory boards for BioCryst, CSL Behring, Novartis, Pharvaris, Swedish Orphan Biovitrum, and Takeda. Dr H. James Wedner reports receiving research support from Allergy Therapeutics, Astria Therapeutics, BioMarin, CSL Behring, Ionis Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, and Takeda; consulting for Astria Therapeutics, BioMarin, CSL Behring, Ionis Pharmaceuticals, KalVista Pharmaceuticals, Pharvaris, and Takeda; and being a speaker for Blueprint Medicines, CSL Behring, GlaxoSmithKline, and Takeda. Dr Daniel Nova Estepan, and Ms Maureen Watt are employees of Takeda Development Center Americas, Inc, and hold stock/options in Takeda Pharmaceutical Company Limited. Dr Irmgard Andresen is an employee of Takeda Pharmaceuticals International, AG, and holds stock/options in Takeda Pharmaceutical Company Limited. Ms Salomé Juethner is an employee of Takeda Pharmaceuticals USA, Inc., and holds stock/options in Takeda Pharmaceutical Company Limited. Dr Natalie Khutoryansky is an employee of Cytel, contracted by Takeda Development Center Americas, Inc. Dr Inmaculada Martinez-Saguer reports receiving research funding support and/or speaker/consultant fees from BioCryst, CSL Behring, KalVista Pharmaceuticals, Octapharma, Pharming, Pharvaris, and Takeda/Shire.
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