Experimental Treatments for Angioedema

Gene Therapy

Gene therapy could be a viable approach for treating hereditary angioedema (HAE), which is caused by genetic mutations in either the SERPING1 gene (types 1 and 2) or the F12 gene (type 3). Gene therapies for HAE are currently in the preclinical stages of development.

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KVD824

KVD824 is an experimental oral medication designed to act as an inhibitor of plasma kallikrein, keeping the enzyme from increasing levels of bradykinin in the body to prevent HAE attacks. The investigational therapy is being tested in Phase 2 trials.

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KVD900

KVD900 is an experimental, oral small-molecule inhibitor of plasma kallikrein. It binds to kallikrein and prevents it from activating bradykinin, potentially reducing or even preventing swelling. Positive top-line results of a Phase 2 trial in HAE patients were recently announced.

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PHA121

PHA121 is an experimental oral, small molecule that binds to the bradykinin B2 receptor, blocking bradykinin from activating it, potentially stopping or preventing HAE attacks. A Phase 2 trial in patients with type 1 or 2 HAE was recently launched.

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SHP616

SHP616 is a type of C1 esterase inhibitor being developed to prevent the acute swelling attacks in HAE patients. It is administered as a subcutaneous (under-the-skin) injection. The experimental therapy completed a Phase 3 trial in 2017.

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Tranexamic Acid

Tranexamic acid, which is approved to enhance blood clotting and is typically used to treat bleeding problems, also may help prevent HAE attacks. However, a Phase 3 trial showed that it did not perform as well as approved therapy Firazyr.

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Xolair

Xolair (omalizumab), which is approved for the treatment of allergic asthma and chronic idiopathic urticaria, is also being investigated for the treatment of angioedema. It has been tested in Phase 3 and Phase 4 trials.

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