People with hereditary angioedema (HAE), a rare genetic disorder, may experience episodes of debilitating, unpredictable, and potentially life-threatening swelling in the abdomen, larynx (voice box), face, and extremities.
Unlike older drugs for hereditary angioedema designed to treat swelling once it happens, Andembry was created to prevent attacks from happening in the first place. The new drug blocks a protein called activated factor 12 (FXIIa) that is responsible for setting off a chain reaction in the body that leads to these episodes.
What Causes Hereditary Angioedema?
People with hereditary angioedema experience sudden swelling because they lack a protein in their blood known as C1 inhibitor (C1-Inh) that’s responsible for controlling inflammation and regulating immune responses, says Bruce Ritchie, MD, a professor of hematology at the University of Alberta in Canada.
“With reduced amounts of this C1-Inh protein, patients can experience unpredictable episodes of swelling anywhere on their body, often precipitated by stress or small traumas,” Dr. Ritchie says. “These swellings can range from merely troublesome to life-threatening if they occur near the patient’s breathing passage or airway.”
Traditional treatments for hereditary angioedema have involved replacing C1-Inh using infusions of this protein from donor blood, Ritchie says. Patients can use these infusions to treat attacks, and they can also give themselves injections under the skin up to three times a week to help prevent attacks, Ritchie says.
One key advantage of Andembry is that its preventive effects may last for two to four weeks, instead of just a handful of days, Ritchie says.
“Garadacimab has a longer half life with excellent efficacy and targets a different pathway, factor 12a,” says Jonathan Bernstein, MD, a professor at the University of Cincinnati College of Medicine who worked on research studies of garadacimab for hereditary angioedema. Plus, he says, “It has very few side effects.”
People Taking Andembry Had Less Than 1 HAE Swelling Attack per Month on Average
These are better outcomes than patients can get with C1-Inh infusions from donor blood, Ritchie says. Many people who routinely use C1-Inh infusions to prevent swelling attacks may switch to garadacimab, Ritchie predicts.
“The approval of garadacimab should hopefully lead to availability to patients with more severe hereditary angioedema, who use C1-Inh [for attack prevention] or frequently on demand,” Ritchie says.