New Nordisk A/S Sponsored ADR Class B (NVO.US) Alhemo subcutaneous injection approved by the FDA to prevent or reduce bleeding frequency in patients aged 12 and above with hemophilia.

Novo Nordisk A/S Sponsored ADR Class B company (Novo Nordisk) (NVO.US) recently announced that the U.S. FDA has approved Alhemo (concizumab) subcutaneous injection as a once-daily prophylactic treatment for preventing or reducing bleeding frequency in adult and pediatric patients with hemophilia A or B aged 12 and older who have inhibitors of clotting factors. Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist, administered via subcutaneous injection. The press release noted that many current treatments for hemophilia A or B patients with inhibitors require intravenous infusions, making Alhemo the first subcutaneous injection treatment option for this population. It is estimated that around 30% of severe hemophilia A patients and 5%-10% of severe hemophilia B patients develop inhibitors of clotting factors, making their hemophilia treatment more challenging. Despite significant improvements in the quality of life for many hemophilia patients with existing treatments, hemophilia B patients with inhibitors still face limited options for prophylactic treatment and carry a burden of disease and treatment. Based on the unmet medical needs in this population and the results of phase 2 clinical trials, the FDA previously granted breakthrough therapy designation to Alhemo for the treatment of hemophilia B patients with inhibitors. This approval is based on the positive results of the pivotal phase 3 clinical trial explorer7. Data shows that patients receiving prophylactic treatment with Alhemo experienced an 86% reduction in annualized bleeding rate (ABR) compared to those not receiving prophylactic treatment. The estimated mean ABR for patients receiving Alhemo prophylactic treatment was 1.7, compared to 11.8 for those not receiving prophylactic treatment. The median ABR in the Alhemo treatment group was zero, while it was 9.8 for those not receiving prophylactic treatment. As a secondary efficacy support endpoint, 64% of patients receiving Alhemo prophylactic treatment did not experience any spontaneous or traumatic bleeds in the first 24 weeks of treatment, compared to 11% of those not receiving prophylactic treatment. In the explorer7 study, the most common adverse reactions among patients receiving Alhemo treatment were injection-site reactions (18%) and urticaria (6%). Serious adverse reactions included kidney infarction and allergic reactions.