Novo Nordisk's Alhemo subcutaneous injection has been approved by the FDA to prevent or reduce the frequency of bleeding in patients aged 12 and older with hemophilia

According to Zhitong Finance APP, Novo Nordisk (NVO.US) recently announced that the U.S. FDA has approved Alhemo (concizumab) subcutaneous injection as a once-daily preventive treatment to prevent or reduce the bleeding frequency in adult and pediatric patients aged 12 years and older with hemophilia A or B who have inhibitors to clotting factors. Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist suitable for subcutaneous injection. The press release noted that many current treatments for hemophilia A or B patients with inhibitors require intravenous infusion, while Alhemo is the first subcutaneous treatment option among similar therapies for this population.

It is estimated that about 30% of patients with severe hemophilia A and 5%-10% of patients with severe hemophilia B will develop inhibitors to clotting factors, making treatment for these patients more challenging. Although existing treatments have significantly improved the quality of life for many hemophilia patients, those with hemophilia B and inhibitors still bear the burden of the disease and treatment due to limited preventive treatment options. Based on the unmet medical needs in this population and the results of Phase 2 clinical trials, the FDA granted Alhemo breakthrough therapy designation 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 showed that the annual bleeding rate (ABR) of patients receiving Alhemo preventive treatment was reduced by 86% compared to those not receiving preventive treatment. The estimated average ABR for patients receiving Alhemo preventive treatment was 1.7, while the ABR for those not receiving preventive treatment was 11.8. The median ABR for the Alhemo treatment group was zero, while the median ABR for patients not receiving preventive treatment was 9.8. As a secondary efficacy support endpoint, 64% of patients receiving Alhemo preventive treatment did not experience any spontaneous or traumatic bleeding within the first 24 weeks of treatment, compared to 11% in the group not receiving preventive 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 renal infarction and allergic reactions