Document is current

Article History

Received: 20 February 2025

Accepted: 25 April 2025

First Online: 12 June 2025

Declarations

:

: Jonathan A. Bernstein has been or is currently a clinical investigator for BioCryst, BioMarin Pharmaceutical, CSL Behring, Ionis, KalVista Pharmaceuticals, Pharming Group, and Takeda; consultant for BioCryst, BioMarin Pharmaceutical, CSL Behring, Ionis, KalVista Pharmaceuticals, Pharming Group, and Takeda; and a member of the Hereditary Angioedema Medical Advisory Board and US Hereditary Angioedema Association. Stephen D. Betschel has been a speaker and/or received advisor fees from Astria Therapeutics, BioCryst, CSL Behring, Ionis, KalVista Pharmaceuticals, Pharvaris, and Takeda; and research funding from CSL Behring and Takeda. 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. Aleena Banerji has received research support from Astria Therapeutics, Ionis, and Takeda; and been an advisory board member for ADARx Pharmaceuticals, Astria Therapeutics, BioCryst, BioMarin Pharmaceutical, CSL Behring, Intellia Therapeutics, KalVista Pharmaceuticals, Pharvaris, and Takeda. H. James Wedner has received research support from Astria Therapeutics, BioCryst, CSL Behring, Ionis, KalVista Pharmaceuticals, Pharming Group, Pharvaris, and Takeda; been a speaker for BioCryst, BioMarin Pharmaceutical, Blueprint Medicines, CSL Behring, KalVista Pharmaceuticals, Pharming Group, and Takeda; and consulted for ADARx Pharmaceuticals, Astria Therapeutics, BioCryst, CSL Behring, Ionis, KalVista Pharmaceuticals, Pharming Group, and Takeda. Michael Manning has been a researcher/primary investigator and speaker/consultant for BioCryst, BioMarin Pharmaceutical, CSL Behring, Ionis, KalVista Pharmaceuticals, Pharming Group, Pharvaris, and Takeda. Rafael H. Zaragoza-Urdaz has been or is currently a clinical investigator for KalVista Pharmaceuticals, Pharvaris, and Takeda; has received honoraria from/been a member of a speaker’s bureau for BioCryst, CSL Behring, Pharming Group, and Takeda; been an advisory board member for BioCryst, Pharming Group, Regeneron, and Takeda; and received research support from AstraZeneca, GlaxoSmithKline, KalVista Pharmaceuticals, and Takeda. John Anderson has received personal fees for steering committee, advisory board, speaker bureau, and/or clinical research activity from BioCryst, BioMarin Pharmaceutical, CSL Behring, Cycle Pharmaceuticals, KabiCare, KalVista Pharmaceuticals, Pharming Group, Pharvaris, and Shire/Takeda. Remi Gagnon has served as a researcher for ALK-Abelló, AstraZeneca, DBV Technologies, GlaxoSmithKline, Moderna, Novartis, Regeneron, Sanofi, and Takeda; been an advisory board member for ALK-Abelló, Bausch & Lomb, Regeneron, Sanofi Genzyme, and Takeda; and received fees for speaker bureaus from Bausch & Lomb and Novartis. Alan P. Baptist reports research support from BioCryst, Ionis, and Takeda. Daniel Soteres is an advisor for CSL Behring, Cycle Pharmaceuticals, KalVista Pharmaceuticals, and Shire/Takeda; consultant for BioCryst, KalVista Pharmaceuticals, and Pharming Group; conducted research for BioCryst, BioMarin Pharmaceutical, Ionis, KalVista Pharmaceuticals, Pharvaris, and Shire/Takeda; and a speaker for BioCryst, CSL Behring, Pharming Group, and Shire/Takeda. William R. Lumry is a member of advisory boards for Astria Therapeutics, BioCryst, BioMarin Pharmaceutical, CSL Behring, Intellia Therapeutics, KalVista Pharmaceuticals, Pharvaris, and Takeda; received research grants from BioCryst, BioMarin Pharmaceutical, CSL Behring, Ionis, KalVista Pharmaceuticals, and Takeda; consulting fees from Astria Therapeutics, BioCryst, CSL Behring, Fresenius Kabi, Pharming Group, and Takeda; payments for lectures from CSL Behring, Pharming Group, and Takeda; and is an advisory board member of the US Hereditary Angioedema Association. Timothy Craig has served as a speaker and researcher for Astria Therapeutics, BioMarin Pharmaceutical, Ionis, CSL Behring, Intellia Therapeutics, KalVista Pharmaceuticals, and Takeda; researcher for Pharvaris; speaker for Grifols; consultant for Astria Therapeutics, BioCryst, BioMarin Pharmaceutical, CSL Behring, Intellia Therapeutics, Ionis, and Takeda; Director for ACARE International Hereditary Angioedema Center; and member of the Medical Advisory Board for the HAE-A. Timothy Craig is an Editorial Board member of Advances in Therapy. Timothy Craig was not involved in the selection of peer reviewers for the manuscript nor any of the subsequent editorial decisions. Daniel Petroni reports advisory boards for Takeda and has been a research/primary investigator for BioCryst and Takeda; he is now an employee of BioCryst. F. Ida Hsu received research support from Takeda; served on advisory boards for BioCryst, CSL Behring, KalVista Pharmaceuticals, and Takeda; was a speaker for Amgen, Pharming, and Shire/Takeda; and is a consultant for Intellia Therapeutics. Daniel Nova Estepan and Maureen Watt are employees of Takeda Development Center Americas, Inc. and hold stock and/or share options in Takeda Pharmaceutical Company Limited. Salomé Juethner is an employee of Takeda Pharmaceuticals USA, Inc., and holds stock and/or share options in Takeda Pharmaceutical Company Limited. Natalie Khutoryansky is an employee of Cytel, contracted by Takeda Development Center Americas, Inc. Bruce L. Zuraw has received consulting fees from Adverum Biotechnologies, BioCryst, CSL Behring, and Takeda.

: EMPOWER was conducted in accordance with the International Council for Harmonisation Good Clinical Practice Guidelines, ethical principles that have their origins in the Declaration of Helsinki, and other local ethical and legal requirements. The study was reviewed and approved in all study sites by a central review board (Western-Copernicus Group [WCG] Institutional Review Board) or institutions’ local ethics committees (Table  in the Supplementary Material). All patients or their legally authorized representatives provided informed consent to participate in the study.