The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A with factor VIII inhibitors or hemophilia B with factor IX inhibitors.
Alhemo is a subcutaneous therapy, which is developed using a synthetically produced (recombinant) antibody derived from a single cell clone, designed to be equally effective in individuals with hemophilia A and B, irrespective of inhibitor status. It targets an anticoagulant protein known as tissue factor pathway inhibitor (TFPI). It works by blocking and effectively preventing TFPI from performing the anticoagulant function that it naturally carries out in the human body. This type of therapy allows treaters to forgo the need for regular prophylaxis with traditional, intravenously infused factor replacement.
“The development of inhibitors remains the most serious treatment-related complication for people living with hemophilia. For patients with inhibitors, especially in hemophilia B, their hemophilia may remain poorly controlled and pose a life-threatening risk,” said Amy Shapiro, MD, CEO and co-medical director at the Indiana Hemophilia & Thrombosis Center, Inc. “The approval of Alhemo® – a first-of-its-kind, prophylaxis, subcutaneous injection pen for adults and children 12 years and older with hemophilia A and B with inhibitors – provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options, ultimately providing more patients with inhibitors the opportunity to personalize their care and address current treatment gaps.”
Visit the FDA website to learn more about Alhemo including the clinical trials that contributed to its approval.
Source: PR Newswire, December 20, 2024
