The US Food and Drug Administration has approved Andembry (garadacimab), a once-monthly injection, to prevent swelling attacks in people who have the rare disorder hereditary angioedema (HAE).
HAE causes swelling attacks that can range from uncomfortable to potentially life-threatening, and Andembry is the first drug to target activated factor 12, a protein responsible for the swelling caused by the condition.
People with HAE may experience episodes of debilitating, unpredictable, and potentially life-threatening swelling in the abdomen, larynx (voice box), face, and extremities, but the new, first-of-its-kind drug, will now help to prevent these attacks, say experts.
Andembry is injected under the skin of adults and children 12 and older.
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.
Everyday Health reports that the medication blocks a protein called activated factor 12 (FXIIa) that is responsible for setting off the chain reaction in the body that leads to these episodes.
“People with HAE now have another choice for lessening the burden associated with this lifelong condition,” said Anthony Castaldo, CEO of the advocacy non-profit organisation US Hereditary Angioedema Association.
Causes of hereditary angioedema
People with HEA ma experience sudden swelling because they lack a protein in their blood known as C1 inhibitor (C1-Inh) which is responsible for controlling inflammation and regulating immune responses, says Bruce Ritchie, MD, a Professor of Haematology 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. 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 can also inject themselves under the skin up to three times a week to help prevent attacks.
One key advantage of Andembry is that its preventive effects may last for two to four weeks, instead of just a handful of days.
“Garadacimab has a longer half life with excellent efficacy and targets a different pathway, factor 12a,” said 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 said, “it has very few side effects.”
In a late-stage clinical trial, researchers randomly assigned 39 people with hereditary angioedema to take garadacimab and 25 people to take placebo injections for six months. During the study period, people taking Andembry had an average of 0.27 swelling attacks per month, compared with an average of 2.01 monthly attacks for people taking the placebo.
The most common treatment side effects were upper-respiratory tract infections, colds, headaches, and stomach pain. About 14% of those taking Andembry reported injection site reactions, including bruising, discoloration, itching, and rash.
These are better outcomes than patients can get with C1-Inh infusions from donor blood, Ritchie said.
Everyday Health article – FDA Approves New Preventive Drug for HAE (Open access)
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