A new drug designed to prevent hereditary angioedema (HAE) attacks has received approval from the U.S. Food and Drug Administration, marking a major advancement in the treatment of this rare but potentially life-threatening condition. The medication, marketed as Andembry, is a first-in-class therapy targeting the root cause of HAE and is the only preventive treatment available with a once-a-month dosing schedule.
HAE is a rare genetic disorder that affects approximately 1 in 50,000 people. It is caused by a deficiency or dysfunction of a protein called C1 inhibitor, which normally plays a role in controlling inflammation. When this protein is not functioning properly, it can trigger episodes of severe swelling in various parts of the body, including the face, abdomen, limbs, and airways. Attacks can be extremely painful and, when affecting the throat or face, can become life-threatening due to the risk of airway obstruction.
The newly approved drug, Andembry, offers a preventive approach for patients aged 12 years and older. It works by targeting a protein known as factor XIIa, which plays a central role in initiating the swelling cascade in people with HAE. By acting at the top of this biological pathway, Andembry prevents the swelling process from beginning at all, offering a more proactive strategy than existing treatments that only address attacks once they have started.
Clinical trials evaluated the effectiveness of Andembry over a six-month period. Participants received either a 400-milligram starting dose followed by 200 milligrams monthly, or a placebo. Results demonstrated that 62% of individuals who received Andembry experienced no attacks during the trial. Across the board, the drug significantly reduced HAE attacks by an average of 89%, and some individuals saw their attacks drop by over 99%. The treatment also decreased the need for emergency interventions and reduced the frequency of moderate to severe attacks by around 90%.
An ongoing study continues to assess the long-term safety and efficacy of the drug, with interim data showing sustained benefits and a favorable safety profile.
Andembry is administered via a monthly subcutaneous injection using an autoinjector, making it accessible for patients or caregivers to use at home. This simplifies treatment and reduces dependence on frequent medical visits.
Reported side effects are generally mild and include throat irritation, nasal congestion, abdominal discomfort, and local injection site reactions such as redness, swelling, itching, or rash.
The approval of Andembry introduces a promising new option for individuals living with HAE, potentially improving their quality of life by offering effective, convenient, and sustained protection against debilitating attacks. The treatment is expected to become available by the end of June.
