CSL Behring Receives Positive IQWiG Assessment for Garadacimab in Germany

Marburg, Germany – 26 June 2025 - CSL Behring Germany announced a milestone in the early benefit assessment of garadacimab by Germany’s Institute for Quality and Efficiency in Health Care (IQWiG).

Garadacimab is a unique once-monthly treatment targeting factor XIIa to prevent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.

IQWiG has acknowledged that, for the first time, suitable data are available in the indication of hereditary angioedema for a comparison with the appropriate comparative therapy. According to the official IQWiG summary, the benefit assessment was based on a methodologically sound indirect comparison using placebo as a common comparator.

IQWiG concludes in its publication, a hint of considerable additional benefit for garadacimab – a first-of-its-kind recognition based on an anchored indirect comparison (Bucher method) using aggregated trial data. This marks a precedent in the AMNOG process since its inception in 2011.

Despite the absence of individual patient data from the comparator study, IQWiG validated the robustness of CSL Behring’s indirect treatment comparison (ITC) against one of three appropriate comparative therapies defined by the G-BA.

Key Highlights from the IQWiG Assessment:

• Reduction in monthly attack rate: Considerable additional benefit
• EQ-VAS (general health status): Minor additional benefit
• AE-QoL (quality of life): Minor additional benefit

CSL Behring submitted data from the VANGUARD study for garadacimab and compared it with the phase 3-studies for an appropriate comparative therapy defined by the G-BA. All studies included a 24-week placebo-controlled treatment phase, meeting the minimum duration required for benefit assessment.

IQWiG emphasized that the statistically significant reduction in HAE attack frequency observed with garadacimab represents a clinically relevant therapeutic advantage. The agency also acknowledged improvements in patient-reported outcomes, including general health status and disease-specific quality of life.

“This outcome represents a significant milestone,” said Verena Donatz, Market Access Head Germany at CSL Behring. “For the first time, IQWiG has recognized an additional benefit based on an indirect comparison without access to individual patient data. It’s a strong validation of our evidence strategy and the dedicated efforts of everyone involved in supporting access to innovative treatment options.”

While this assessment is a critical first step, the process continues with the final G-BA decision anticipated in late August 2025. CSL Behring remains committed to engaging with stakeholders throughout this process and will continue to provide updates as the
G-BA decision approaches.