Concizumab-mtci Gets Nod for Hemophilia A, B Prophylaxis in Patients With Inhibitors

Officials with the FDA have granted the anti–tissue factor pathway inhibitor (TFPI) monoclonal antibody concizumab-mtci (Alhemo; Novo Nordisk) approval to be used for hemophilia A and B prophylaxis in patients who are ≥12 with hemophilia A factor VIII inhibitors or hemophilia B factor IX inhibitors.¹

The approval followed a call for additional monitoring and manufacturing information from the agency in April 2023. They based their approval on data from the open-label, parallel-group, phase 3 EXPLORER 7 trial (NCT04083781), where concizumab was shown to significantly reduce the estimated mean annualized bleeding rate (ABR) compared with no prophylaxis in 91 adult and 42 adolescent male patients (1.7 vs 11.8, respectively; ABR ratio, 0.14; P<.001).

According to an FDA statement, “the most common adverse reactions (reported in at least 5% of patients) to Alhemo were injection site reactions and hives. Hypersensitivity reactions including redness of skin (erythema), rash, itching (pruritus), and abdominal pain have occurred in patients treated with Alhemo…treatment with Alhemo may increase the risk of blood clots.”

The approval makes concizumab the second anti-TFPI antibody available in the United States, following the approval of marstacimab-hncq (Hympavzi; Pfizer) in October 2024 for hemophilia A or B without inhibitors.²

“The development of inhibitors remains the most serious treatment-related complication for people living with hemophilia. For patients with inhibitors, especially in hemophilia B, their hemophilia may remain poorly controlled and pose a life-threatening risk,” said Amy Shapiro, MD, CEO and co-medical director at the Indiana Hemophilia & Thrombosis Center, Inc. in a statement from Novo. “The approval of Alhemo—a first-of-its-kind, prophylaxis, subcutaneous injection pen for adults and children 12 years and older with hemophilia A and B with inhibitors—provides a much-needed alternative to the current standard of care in hemophilia B with inhibitors, while offering patients with hemophilia A with inhibitors more treatment options, ultimately providing more patients with inhibitors the opportunity to personalize their care and address current treatment gaps.”³

References

1. FDA. FDA approves drug to prevent or reduce the frequency of bleeding episodes for patients with hemophilia A with inhibitors or hemophilia B with inhibitors. Press release. Published December 20, 2024. Accessed January 2, 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-prevent-or-reduce-frequency-bleeding-episodes-patients-hemophilia-inhibitors-or
2. Pfizer US FDA approves Pfizer’s Hympavzi (marstacimab-hncq) for the treatment of adults and adolescents with hemophilia A or B without inhibitors. Press release. Published October 11, 2024. Accessed January 2, 2025. https://www.businesswire.com/news/home/20241009505480/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-HYMPAVZI%E2%84%A2-marstacimab-hncq-for-the-Treatment-of-Adults-and-Adolescents-with-Hemophilia-A-or-B-Without-Inhibitors
3. Novo. FDA approves Alhemo injection 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 with inhibitors. Press release. Published December 20, 2024. Accessed January 2, 2025. https://www.prnewswire.com/news-releases/fda-approves-alhemo-injection-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-with-inhibitors-302337790.html