Global biotechnology leader CSL recently announced the FDA’s approval of ANDEMBRY, a monthly subcutaneous (under-the-skin) self-injection. The treatment was recently FDA approved for the prevention of hereditary angioedema (HAE) attacks in patients twelve years of age or older.

According to an article in PR Newswire, the approval was based on the results of the VANGUARD clinical trial (NCT04656418). Its method of self-injection is convenient when compared to prior therapies requiring either intravenous infusions or an increased number of injections.

Factor Xlla, a plasma protein, is ANDEMBRY’s target. It is influential in promoting the series of interconnected biological events that lead to HAE attacks. The therapy is a novel approach in managing the disorder. Targeting factor Xlla which causes attacks of swelling in HAE patients is instrumental in inhibiting HAE attacks. The self-injection is delivered within fifteen seconds by way of an autoinjector containing a citrate-free formula.

HAE attacks can be painful, affecting various areas of the body, and occurs in approximately 1 in 50,000 people.

Significant Reductions

Results of the Clinical trial showed as much as a ninety-nine percent reduction in HAE attacks when treated with ANDEMBRY. Sixty-two percent of recipients continued to be free of attacks during the entire study period.

About HAE

This genetic disorder has the potential to be life-threatening and is characterized by unpredictable and recurring attacks of angioedema. An interim analysis, published in Allergy Oct. 2024 spoke of ANDEMBRY’S favorable long-term safety profile and reductions in HAE attacks.

The Safety Profile is Favorable

The most common adverse events pertained to injection site reactions. It also included nasopharyngitis and abdominal pain. The approval allows HAE patients to have a new option to control the disease over the long-term.

CLS Behring has announced that ANDEMBRY will launch commercially by the end of June of this year.