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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

PLAINSBORO, N.J., July 31, 2025 /PRNewswire/ -- Novo Nordisk announced today that the US Food and Drug Administration (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.1,2,3 Currently, many treatments for HA/HB without inhibitors are administered via intravenous infusions.2 With this approval, Alhemo now offers a subcutaneous injection treatment option for this population.1

"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.4,5

"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).2 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%).1

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.

About the explorer8 study
Explorer8 is a phase 3 clinical trial that evaluated the efficacy and safety of Alhemo® for adults and pediatric patients 12 years of age and older living with hemophilia A or B (HA/HB) without inhibitors.2 In explorer8, among the 156 males with HA/HB included in the trial, 63 were randomly assigned in a 1:2 ratio to receive no prophylaxis (arm 1, n=21), or Alhemo prophylaxis (arm 2, n=42).2,6 The median duration of treatment was 24.1 weeks in arm 1 (no prophylaxis) and 32.1 weeks in arm 2 (Alhemo prophylaxis).The initial loading dose of Alhemo was 1 mg per kilogram of body weight, followed by 0.2 mg per kilogram daily, and potentially individualized on the basis of concizumab-mtci plasma concentration as measured at week 4.1,2Efficacy was evaluated separately in HA patients without inhibitors and in HB patients without inhibitors when all patients in arms 1 and 2 had completed the main part of the trial (at least 24 or at least 32 weeks, respectively), based on the number of treated bleeding episodes comparing Alhemo prophylaxis and no prophylaxis.1

About Alhemo (concizumab-mtci)
Alhemo is a tissue factor pathway inhibitor (TFPI) antagonist. By inhibiting the TFPI, Alhemo enhances factor Xa (FXa) production during the initiation phase of coagulation, leading to improved thrombin generation and clot formation in patients with hemophilia A or B (HA/HB) with or without inhibitors. The effect of Alhemo is not influenced by the presence of inhibitory antibodies to FVIII or FIX and Alhemo does not induce or enhance the development of direct inhibitors to FVIII or FIX. Alhemo is approved as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and pediatric patients 12 years of age and older with HA/HB with or without inhibitors in the US.1

What is Alhemo?
Alhemo (concizumab-mtci) injection 60 mg, 150 mg, or 300 mg is a prescription medicine used for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children 12 years of age and older with hemophilia A or B with or without factor VIII or IX inhibitors.

Important Safety Information

What is the most important information I should know about Alhemo?

Do not use Alhemo if you are allergic to concizumab-mtci or any of the ingredients in Alhemo.

Before using Alhemo, tell your HCP about all of your medical conditions, including if you:

Tell your HCP about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and supplements.

What are the possible side effects of Alhemo?
Alhemo may cause serious side effects, including:

The most common side effects of Alhemo include: bruising, redness, bleeding, itching, rash or lump at the injection site, headache, and hives. These are not all the possible side effects of Alhemo. Call your doctor for medical advice about side effects.

About hemophilia
Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.7 It is estimated to affect approximately ~800,000 people worldwide.8 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing. Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and hemophilia B is caused by a missing or defective clotting Factor IX (FIX).7 Hemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy.8

About Novo Nordisk
Novo Nordisk is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for more than 100 years. This heritage has given us experience and capabilities that also enable us to drive change to help people defeat other serious chronic diseases such as obesity, rare blood, and endocrine disorders. We remain steadfast in our conviction that the formula for lasting success is to stay focused, think long-term, and do business in a financially, socially, and environmentally responsible way. With a US presence spanning 40 years, Novo Nordisk US is headquartered in New Jersey and employs over 10,000 people throughout the country across 12 manufacturing, R&D, and corporate locations in eight states plus Washington DC. For more information, visit novonordisk-us.com, Facebook, Instagram, and X.

References

  1. Alhemo (concizumab-mtci) injection, for subcutaneous use [package insert]. Plainsboro, NJ: Novo Nordisk Inc.
  2. Chowdary P., et al. Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial. Lancet Hematology. 2024;11:e891-904.
  3. Novo Nordisk. FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors. Accessed May 2025. Available at https://www.novonordisk-us.com/media/news-archive/news-details.html?id=915084.
  4. Shapiro AD. Concizumab: a novel anti-TFPI therapeutic for hemophilia. Blood Adv. 2021;5(1):279.
  5. Matsushita T, Shapiro A, Abraham A, et al. Phase 3 trial of concizumab in hemophilia with inhibitors. N Engl J Med. 2023; 389(9): 783-794.
  6. ClinicalTrials.gov. Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors (explorer8). Accessed May 2025. Available at https://clinicaltrials.gov/study/NCT04082429.
  7. MedlinePlus. Hemophilia. Accessed May 2025. Available at https://medlineplus.gov/genetics/condition/hemophilia/.
  8. World Federation of Hemophilia. Annual Global Survey 2023. Accessed July 2025. https://wfh.org/research-and-data-collection/annual-global-survey/.

SOURCE Novo Nordisk

Posted: July 2025

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