Alhemo: A Breakthrough in Hemophilia Treatment – FDA Approves Novo Nordisk’s Once-Daily Prophylactic Injection for Hemophilia A and B with Inhibitors

FDA Approval:
The U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) as a once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes in adults and children aged 12 years and older with hemophilia A or B with inhibitors1 3 4.

Mechanism of Action:
Alhemo is an anti-tissue factor pathway inhibitor (TFPI) monoclonal antibody that blocks TFPI, a protein that stops blood from clotting, thereby ensuring the production of thrombin, which helps to clot the blood and prevent bleeding even in the presence of inhibitors1 2 4.

Clinical Trial Results:
The pivotal Phase 3 explorer7 study showed an 86% reduction in annual bleeding rate (ABR) in patients randomized to receive Alhemo, demonstrating its efficacy in preventing bleeding episodes1 3.

Unmet Medical Need:
The approval addresses a significant unmet need for patients with hemophilia A or B with inhibitors, who often have limited treatment options and face a higher risk of poorly controlled bleeding episodes1 2 4.

Global Approvals:
Alhemo is also approved in Australia, Japan, Switzerland, and the EU, with specific indications varying by country1 2 5.

Breakthrough Therapy Designation:
The FDA granted Breakthrough Therapy designation to Alhemo based on results from Phase 2 clinical trials, highlighting its potential to address critical needs in hemophilia treatment1 4.

Treatment Administration:
Alhemo is administered via a prefilled, premixed pen for subcutaneous injection, making it a more convenient and less invasive treatment option compared to traditional intravenous infusions1 3 4.