FDA Approves Concizumab as Daily Prophylaxis for Hemophilia A and B
Many treatments for hemophilia A and B are administered via intravenous infusions, so the approval offers a subcutaneous injection for the treatment for patients 12 years and older.
The FDA approved concizumab-mtci (Alhemo) as a once daily subcutaneous prophylaxis injection to prevent or reduce the frequency of bleeding episodes for patients 12 years and older with hemophilia A or B without inhibitors. Currently, many treatments for these disease states are administered via intravenous infusions, so this approval now offers a subcutaneous injection for the treatment of this patient population.1
Many treatments for hemophilia A and B are administered via intravenous infusions, so the approval offers a subcutaneous injection for the treatment for patients 12 years and older. | Image Credit: PhotoGranary - stock.adobe.com
"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," Anna Windle, PhD, senior vice president of clinical development and medical and regulatory affairs at Novo Nordisk, said in a news release.1
In a study published in Haemophilia, community pharmacy involvement in dispensing hemophilia treatment improved the benefits and outcomes for patients. The investigators found that door-to-door travel times saved approximately 16.5 minutes for the patient. Furthermore, patients were mostly satisfied with their overall satisfaction, travel time, and relationship with their community pharmacist compared to the hospital pharmacy.2
The approval was based on the explorer8 (NCT04082429), with investigators determining the efficacy of concizumab and how it works without inhibitors for hemophilia A or B. Investigators included patients who usually take medicine to treat bleeds on demand and placed individuals into 2 groups: one where patients received the study medication from the start of the study and one where patients started the study medication after 6 months. Concizumab was injected under the skin once daily and could be used at home, and the study lasted up to 6.5 years. Patients visited the clinic every 4 weeks for the first 6 to12 months, then every 8 weeks for the remainder of the study. The primary end point included the number of treated spontaneous and traumatic bleeding episodes, and the secondary outcomes included the number of treated spontaneous bleeding episodes, treated spontaneous and traumatic joint bleeds, thromboembolic events, hypersensitivity-type reactions, and injection site reactions.3
The study results showed a significant reduction in annualized bleeding rates of 79% for patients with hemophilia B and 86% for hemophilia A. Further, there were reductions in average and median ABRs, strengthening the efficacy of the drug in reducing bleeds across patient groups at 3.1 and 1.6, respectively, for hemophilia B and 2.7 and 2.9, respectively, for hemophilia A. The most common adverse reactions reported included injection site reactions and headache.1
"For people living with hemophilia, it is important to continually monitor and discuss bleed control with their healthcare professional," Allison P. Wheeler, MD, scientific director of Washington Center for Bleeding Disorders in Seattle, Washington, said in a news release.1 "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."
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REFERENCES
1. FDA approves Alhemo as once-daily prophylactic treatment to prevent or reduce the frequency of bleeding episodes for adults and children 12 years of age and older with hemophilia A or B (HA/HB) without inhibitors. News release. Novo Nordisk. July 31, 2025. Accessed August 1, 2025. https://prnmedia.prnewswire.com/news-releases/fda-approves-alhemo-as-once-daily-prophylactic-treatment-to-prevent-or-reduce-the-frequency-of-bleeding-episodes-for-adults-and-children-12-years-of-age-and-older-with-hemophilia-a-or-b-hahb-without-inhibitors-302519177.html
2. Chamouard V, Freyssenge J, Duport G, et al. Evaluation of the care pathway in the context of the dispensing of emicizumab (Hemlibra) in community and hospital pharmacies in France: A patient satisfaction survey. Haemophilia. 2023;29(6):1490-1498. doi:10.1111/hae.14857
3. Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8). ClinicalTrials.gov identification: NCT04082429. Updated May 25, 2025. Accessed August 1, 2025. https://clinicaltrials.gov/study/NCT04082429
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