Novo Nordisk announced that the FDA approved Alhemo® (concizumab-mtci) injection as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or B (HA/HB) without inhibitors, expanding on the December 2024 approval for HA/HB with inhibitors. Currently, many treatments for HA/HB without inhibitors are administered via intravenous infusions. With this approval, Alhemo® now offers a subcutaneous injection treatment option for this population.
"The FDA approval of an expanded indication for Alhemo® marks a meaningful step forward for people with hemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option," said Anna Windle, PhD, Senior Vice President, Clinical Development, Medical & Regulatory Affairs, Novo Nordisk. "By building on the initial indication for Alhemo® for those with hemophilia with inhibitors—an especially significant development in hemophilia B where challenges still exist despite advanced treatment options—Novo Nordisk continues its 35+ year legacy in rare bleeding disorders to continue to help address the unmet needs of this community."
Alhemo® is designed to block a protein called tissue factor pathway inhibitor (TFPI), which stops blood from clotting. By inhibiting TFPI, Alhemo® improves the production of thrombin, which helps to clot the blood and prevent bleeding, when clotting factors VIII and IX are missing or deficient regardless of inhibitor status.
"For people living with hemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional," said Allison P. Wheeler, MD, Scientific Director, Washington Center for Bleeding Disorders, Seattle, WA. "With today's approval of Alhemo® for hemophilia A or B without inhibitors, more people living with these rare blood disorders now have a daily prophylaxis option that may help decrease their bleeding rates."
The primary objective of the pivotal phase 3 explorer8 trial was to compare the number of treated spontaneous and traumatic bleeding episodes, as measured by the ABR, in patients aged 12 years and older with HA/HB without inhibitors, receiving Alhemo® prophylaxis versus no prophylaxis (on demand Factor VIII/Factor IX treatment). The study showed a statistically significant reduction in ABR of 79% for HB patients (ABR ratio: 0.21; 95% CI: 0.10–0.45; p<0.0001) and 86% for HA patients (ABR ratio: 0.14; 95% CI: 0.07–0.29; p<0.0001). Additionally, notable reductions in the average and median ABRs further reinforced the efficacy of Alhemo® prophylaxis in reducing bleeds across both patient groups, with an average ABR of 3.1 (1.9-5.0) and a median ABR of 1.6 (0.0-4.8) for HB patients, compared to 14.8 (8.1-26.9) and 14.9 (3.3-22.1), respectively, and an average ABR of 2.7 (1.6-4.6) and median ABR of 2.9 (0.0-5.2) for HA patients, compared to 19.3 (11.3-33.0) and 19.6 (17.3-30.4), respectively. In the study, the most common adverse reactions reported in ≥5% of patients randomized to receive Alhemo® were injection site reactions (7%) and headache (7%).
Alhemo® is provided in prefilled, premixed pens for subcutaneous injection (60 mg/1.5 mL, 150 mg/1.5 mL, or 300 mg/3 mL) via thin 32 gauge, 4 mm needles, which are provided separately, offering an additional treatment choice for patients with hemophilia with inhibitors – and now without inhibitors – to further customize their care.
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