Emicizumab
| Monoclonal antibody | |
|---|---|
| Type | Whole antibody |
| Source | Humanized |
| Target | Activated factor IX, factor X |
| Clinical data | |
| Trade names | Hemlibra |
| Other names | ACE910, RG6013, emicizumab-kxwh |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a622046 |
| License data |
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| Pregnancy category |
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| Routes of administration | Subcutaneous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| ChemSpider |
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| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C6434H9940N1724O2047S45 |
| Molar mass | 145639.02 g·mol−1 |
Emicizumab, sold under the brand name Hemlibra, is a humanized bispecific monoclonal antibody for the treatment of haemophilia A, developed by Genentech and Chugai (both organizations are subsidiaries of Hoffmann-La Roche).[4] Emicizumab is a bispecific factor IXa- and factor X-directed antibody.[3]
In November 2017, it was approved in the United States for treatment of haemophilia A in those who had developed resistance to other treatments.[5] It was subsequently approved by the US FDA in April 2018, under the breakthrough therapy designation for the treatment of haemophila A in those who have not developed resistance to other treatments.[6] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[7]
Medical uses
Emicizumab is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors.[3]
Adverse effects
The most common adverse reactions (incidence ≥10%) are injection site reactions, headache, and arthralgia.[3]
Mechanism of action
Emicizumab binds to both the activated coagulation factor IX and to factor X, mediating the activation of the latter. This is normally the function of coagulation factor VIII, which is missing in haemophilia A patients.[4][8]
Research
A Phase I clinical trial found that it was well tolerated by healthy subjects.[9]
Studies indicate that emicizumab is a better therapy compared to the previous generations, due to subcutaneous administration and fewer injections, which reduces injection site reactions and makes therapy less troublesome.[10]
References
- ^ a b "AUSTRALIAN PRODUCT INFORMATION – Hemlibra (Emicizumab)". Archived from the original on 15 May 2023. Retrieved 8 January 2023.
- ^ "Summary Basis of Decision (SBD) for Hemlibra". Health Canada. 23 October 2014. Retrieved 29 May 2022.
- ^ a b c d "Hemlibra- emicizumab injection, solution". DailyMed. 28 October 2024. Retrieved 11 July 2025.
- ^ a b Spreitzer H (4 July 2016). "Neue Wirkstoffe - Emicizumab". Österreichische Apothekerzeitung (in German) (14/2016).
- ^ "Roche hemophilia drug wins FDA nod, with a warning". Reuters. 17 November 2017.
- ^ "FDA Grants Roche Breakthrough Therapy Designation on Hemophilia Drug". BioPharm International. UBM. 19 April 2018. Retrieved 20 April 2018.
- ^ New Drug Therapy Approvals 2017. U.S. Food and Drug Administration (FDA) (Report). January 2018. Archived from the original (PDF) on 14 September 2019. Retrieved 16 September 2020.
- ^ Shima M, Hanabusa H, Taki M, Matsushita T, Sato T, Fukutake K, et al. (May 2016). "Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A". The New England Journal of Medicine. 374 (21): 2044–53. doi:10.1056/NEJMoa1511769. PMID 27223146.
- ^ Uchida N, Sambe T, Yoneyama K, Fukazawa N, Kawanishi T, Kobayashi S, et al. (March 2016). "A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects". Blood. 127 (13): 1633–41. doi:10.1182/blood-2015-06-650226. PMC 4817308. PMID 26626991.
- ^ Grabowska K, Grzelak M, Zhao LY, Płuciennik E, Pasieka Z, Kciuk M, et al. (May 2024). "Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials". Current Protein & Peptide Science. 25 (9): 719–737. doi:10.2174/0113892037294674240509094418. PMID 38797909.