Garadacimab recommended for approval in Europe to treat HAE
Monthly injection designed to prevent swelling attacks in adolescents, adults
A European Medicines Agency (EMA) committee has recommended that the antibody-based therapy garadacimab be approved as a once-monthly preventive treatment to reduce the risk of swelling attacks in people with hereditary angioedema (HAE) ages 12 and older.
The positive opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) will now be reviewed by the European Commission, which has final say on therapy approvals in the European Union. The commission isn’t required to abide by the CHMP’s recommendation, but it almost always does.
A decision from the European Commission is now expected in the first quarter of 2025, garadacimab developer CSL Behring said in a company press release. If garadacimab is approved in Europe, the company plans to market it under the brand name Andembry. Garadacimab is also being considered for potential approval in the U.S.
“This CHMP decision brings us closer to offering an innovative treatment to patients living with HAE, which is a debilitating and potentially life-threatening condition,” said Emmanuelle Lecomte-Brisset, senior vice president and head of Global Regulatory Affairs at CSL. “We look forward to making this therapy available to patients in Europe.”
Garadacimab now being tested for long-term safety, effectiveness
HAE is caused by genetic mutations that lead to the overproduction of a signaling molecule called bradykinin, which triggers swelling in the deeper layers of the skin or in mucus membranes. Bradykinin is produced by an enzyme called kallikrein. Kallikrein, in turn, is formed when a clotting protein known as factor XII, or FXII, is activated.
Garadacimab is designed to block the activation of FXII, thereby shutting down this chain of molecular events and ultimately reducing the risk of swelling. The therapy would be given once per month, via subcutaneous, or under-the-skin, injection.
The CHMP’s positive recommendation was based on data from the Phase 3 VANGUARD trial (NCT04656418), which tested the therapy against a placebo in 64 HAE patients ages 12 and older. Its results showed that garadacimab reduced swelling attack rates by more than 85%, with two-thirds of patients completely free from swelling attacks over the course of the six-month study.
The trial’s ongoing extension study (NCT04739059), now collecting data on the long-term safety and efficacy of garadacimab, also backed the committee’s positive opinion. That study enrolled more than 160 patients, including some who had participated in VANGUARD or a previous Phase 2 trial (NCT03712228) of garadacimab. All participants in the extension study are being given monthly injections of garadacimab.
A recent interim analysis showed that, with most patients having been followed for longer than one year, garadacimab reduced swelling attack rates by 95%, and more than half of patients were free from swelling attacks. The most common side effects of garadacimab reported in the study were reactions at the injection site.
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