In a significant advancement for patients with hemophilia A and B, the U.S. Food and Drug Administration (FDA) has approved Alhemo (concizumab-mtci) as a once-daily prophylactic treatment to prevent or reduce bleeding episodes in patients over 12 years of age. Manufactured by Novo Nordisk, Alhemo offers a groundbreaking solution for those with hemophilia who have developed inhibitors, a challenging complication that hinders the effectiveness of traditional treatments.

Alhemo is the first treatment of its kind for this population, as it is administered through a subcutaneous injection, a method less invasive than the conventional intravenous infusions. The drug is a tissue factor pathway inhibitor (TFPI) antagonist, designed to block a protein in the body that typically halts blood clotting. By inhibiting TFPI, Alhemo enhances the production of thrombin, a protein essential for blood clotting, helping to prevent bleeding in patients whose clotting factors are missing or deficient.

The approval follows promising results from the Phase 3 explorer7 study, which demonstrated a dramatic 86% reduction in the annual bleeding rate (ABR) for patients receiving Alhemo prophylaxis compared to those who did not. In the study, 64% of patients on Alhemo experienced zero treated spontaneous or traumatic bleeds during the first 24 weeks, a stark contrast to just 11% in the no-prophylaxis group. These findings underscore the efficacy of Alhemo in significantly reducing bleeding episodes in this high-risk population.

“The approval of Alhemo marks a milestone in hemophilia care, providing a much-needed alternative for patients who have limited options due to inhibitors,” said Anna Windle, SVP Clinical Development at Novo Nordisk. “This breakthrough treatment underscores our commitment to developing innovative solutions for rare bleeding disorders.”

Hemophilia, a rare bleeding disorder, affects around 32,000 people in the U.S. alone. The condition impairs the body's ability to form blood clots, leading to frequent and potentially life-threatening bleeding episodes. Hemophilia A is caused by a deficiency of factor VIII, while hemophilia B results from a deficiency of factor IX. In some cases, the body develops inhibitors that neutralize the clotting factors used in treatment, complicating management and increasing the risk of uncontrolled bleeding.

Dr. Amy Shapiro, CEO of the Indiana Hemophilia & Thrombosis Center, emphasized the significance of Alhemo for those with inhibitors, particularly in hemophilia B, where treatment options are limited. “Alhemo offers a new level of care, allowing patients to manage their condition more effectively and safely,” she noted.

Alhemo is already approved in several countries, including Australia, Japan, Switzerland, and the European Union. Its availability in the U.S. represents a major step forward in the treatment of hemophilia, offering patients with inhibitors a promising new option to manage their condition.