Name:
Can new treatment garadacimab prevent attacks in patients with hereditary angioedema?
Description:
Can new treatment garadacimab prevent attacks in patients with hereditary angioedema?
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Duration:
T00H02M31S
Embed URL:
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Content URL:
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Upload Date:
2024-11-07T03:38:21.0561269Z
Transcript:
Language: EN.
Segment:0 .
Hereditary angioedema (HAE) is a rare genetic disease. It causes unpredictable and sometimes severe swelling, called HAE attacks, in different parts of the body. A new medicine called garadacimab is being tested in clinical studies to determine whether it can prevent HAE attacks and to determine its safety profile. Activated factor XII is a blood protein that is the principal initiator of the contact system, ultimately regulating the kallikrein-kinin system and the intrinsic coagulation pathway and it is also involved in the fibrinolytic and the complement pathways.
These pathways play important roles in inflammation and blood coagulation. When the kallikrein-kinin system is over-activated, a build-up of a protein called bradykinin leads to edema (or swelling) and HAE attacks. Garadacimab is an antibody (a specialised protein) that works differently to currently available HAE therapies by blocking the function of activated factor XII. It's injected just under the skin once a month. The VANGUARD study looked at how well garadacimab worked for preventing HAE attacks and whether it caused any side effects.
In this study, patients took either garadacimab or a placebo for 6 months. Compared with patients that received placebo, patients that received garadacimab experienced substantially fewer HAE attacks per month. They were also less likely to suffer moderate or severe attacks, and fewer patients in this group needed treatment with on-demand therapy compared with the placebo group. What was the experience of the patients? 82% of patients treated with garadacimab rated their experience as “good” or “excellent” versus 33% of patients treated with placebo.
Almost two thirds of patients receiving garadacimab remained attack free, while all of the placebo group continued to have HAE attacks. Both groups showed similar proportions of side effects. Overall, the results mean that garadacimab is a potential new treatment to prevent HAE attacks.
