Novo Nordisk reported that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a favorable opinion, suggesting that the label for Alhemo® (concizumab) be updated to cover the treatment of moderate or severe hemophilia B without inhibitors as well as severe hemophilia A.
The findings of the phase 3 Explorer trial, which achieved its primary goal, support the positive CHMP conclusion. For patients with hemophilia A without inhibitors and hemophilia B without inhibitors, the results showed that Alhemo® prophylaxis reduced treated spontaneous and traumatic bleeding by 86% and 79%, respectively, when compared to no prophylactic treatment. Alhemo® demonstrated a favorable safety profile in this trial for patients without inhibitors who had hemophilia A and B.
If approved by the European Commission, this label update would extend Alhemo®’s convenient once-daily, under-the-skin administration to patients living with haemophilia without inhibitors, providing an efficacious prophylactic choice.
Each person living with haemophilia has individual and evolving needs. With its user-friendly, pre-filled, portable pen, we believe that Alhemo® has the potential to offer even more patients an individualised and flexible treatment.
Martin Holst Lange
In particular, trends favoring Alhemo® over prophylaxis were seen in the short-form health survey (SF-36v2) and Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) results for patients with haemophilia A and haemophilia B; these included the change in “bodily pain” and “physical functioning” from baseline to week 24 in the survey, as well as the Haem-A-QoL “total score” and the “physical health” domain score. Treatment preference results showed patients were also in favor of Alhemo® over no prophylaxis or prior treatment, with 70.9% of respondents stating they prefer Alhemo® over their previous haemophilia treatment.
About Alhemo® (concizumab)
Tissue factor pathway inhibitors (TFPIs) are the target of the humanized monoclonal IgG4 antibody concizumab. Clotting factors (Factor VIII in hemophilia A and Factor IX in hemophilia B) are lacking in people with hemophilia. Factor Xa, which is essential for the production of blood clots, is normally inhibited by TFPI. Concizumab improves the function of Factor Xa by blocking TFPI, which increases thrombin production and encourages the formation of clots. Even in patients who have developed inhibitors against conventional factor replacement therapy, this method remains successful.
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Graduated from the University of Kerala'20 with B.Sc. Botany & Biotechnology. Post-graduation in Biotechnology from the University of Kerala'22. Internship experience in Cancer Research.
