CSL Behring’s therapy for hereditary angioedema receives positive assessment in Germany
Ella Day | June 26, 2025 | News story | Market Access, Market Research, Medical Communications, Research and Development, Sales and Marketing | CSL Behring, Institute for Quality and Efficiency in Health Care, Rare Diseases, clinical trials, hereditary angioedema, market access
CSL Behring has announced a key milestone for its hereditary angioedema (HAE) therapy, garadacimab, following a positive benefit assessment by Germany’s Institute for Quality and Efficiency in Health Care (IQWiG). The agency concluded there is a “hint of considerable additional benefit” for garadacimab compared to existing HAE prophylaxis therapies, based on robust indirect comparisons using placebo-controlled data.
Garadacimab is a subcutaneous treatment targeting activated factor XII (FXIIa), offering a new mechanism in the prevention of HAE attacks. The IQWiG opinion is the first to establish an added benefit for an HAE therapy through an indirect comparison, despite the absence of individual patient data for the comparator treatment.
HAE is a rare, genetic condition caused by a deficiency or dysfunction of the C1 inhibitor protein. It leads to unpredictable and painful swelling episodes and if left untreated, attacks can be debilitating or fatal.
The assessment follows the phase 3 VANGUARD study, in which garadacimab demonstrated a reduction in monthly attack rates, along with improvements in general health and quality of life. IQWiG characterised the reduction in HAE attack frequency as a “considerable” benefit, with “minor” additional benefits for the quality-of-life measures.
Verena Donatz, market access head of Germany at CSL Behring, commented: “IQWiG’s recognition validates our evidence strategy and underlines the importance of garadacimab as a treatment option.”
A final reimbursement decision from the Federal Joint Committee is expected by late August.
Garadacimab has recently received approval in multiple countries including the US, UK, EU and Japan.
Ella Day
26/6/25
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