Novo Nordisk (NVO, Financial) has announced that the U.S. FDA has approved Alhemo (concizumab) for subcutaneous injection as a once-daily preventive treatment for hemophilia A or B patients aged 12 and older. These patients have coagulation factor inhibitors. Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist and is the first subcutaneous treatment option for this population, which traditionally requires intravenous infusion.
Approximately 30% of severe hemophilia A patients and 5%-10% of severe hemophilia B patients develop coagulation factor inhibitors, complicating treatment. Despite existing therapies improving quality of life, hemophilia B patients with inhibitors face limited preventive options. The FDA granted Alhemo breakthrough therapy designation based on unmet needs and positive Phase 2 trial results.
Approval was based on the successful Phase 3 trial, explorer7, where Alhemo reduced annual bleeding rates (ABR) by 86% compared to no treatment. The estimated average ABR for Alhemo-treated patients was 1.7, compared to 11.8 for untreated patients. The most common side effects were injection site reactions (18%) and urticaria (6%), with serious reactions including renal infarction and allergic responses.
