Emicizumab

Monoclonal antibody

US DailyMed: Emicizumab
US FDA: Emicizumab

Pregnancy
category

AU: B2^[1]

Routes of
administrationSubcutaneousATC code

B02BX06 (WHO)

Legal statusLegal status

AU: S4 (Prescription only)^[1]
CA: ℞-only / Schedule D^[3]
US: WARNING^[2]Rx-only
EU: Rx-only
In general: ℞ (Prescription only)

IdentifiersCAS Number

1610943-06-0

DrugBank

DB13923

ChemSpider

none

UNII

7NL2E3F6K3

KEGG

D10821^Y

Chemical and physical dataFormulaC₆₄₃₄H₉₉₄₀N₁₇₂₄O₂₀₄₇S₄₅Molar mass145639.02 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 (a subsidiary of Hoffmann-La Roche).^[4] A Phase I clinical trial found that it was well tolerated by healthy subjects.^[5]

In November 2017, it was approved in the United States for treatment of haemophilia A in those who had developed resistance to other treatments.^[6] It was subsequently approved by the US FDA in April 2018 under the breakthrough therapy designation for treatment of haemophila A in those who have not developed resistance to other treatments.^[7] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[8]

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.^[9]

Adverse effects

The most common adverse reactions (incidence ≥10%) are injection site reactions, headache, and arthralgia.^[10]

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]^[11]

References

^ ^a ^b "AUSTRALIAN PRODUCT INFORMATION – Hemlibra (Emicizumab)". Archived from the original on 15 May 2023. Retrieved 8 January 2023.
^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
^ "Summary Basis of Decision (SBD) for Hemlibra". Health Canada. 23 October 2014. Retrieved 29 May 2022.
^ ^a ^b Spreitzer H (4 July 2016). "Neue Wirkstoffe - Emicizumab". Österreichische Apothekerzeitung (in German) (14/2016).
^ Uchida N, Sambe T, Yoneyama K, Fukazawa N, Kawanishi T, Kobayashi S, Shima M (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.
^ "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 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2018. Retrieved 16 September 2020.
^ Grabowska, Katarzyna; Grzelak, Michalina; Zhao, Lin-Yong; Płuciennik, Elżbieta; Pasieka, Zbigniew; Kciuk, Mateusz; Gielecińska, Adrianna; Smakosz, Aleksander K.; Kałuzińska-Kołat, Żaneta; Kołat, Damian. "Emicizumab as a Promising Form of Therapy for Type A Hemophilia - A Review of Current Knowledge from Clinical Trials". Current Protein & Peptide Science. 25: 1–18. doi:10.2174/0113892037294674240509094418.
^ FDA Professional Drug Information
^ 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.

External links

"Emicizumab". Drug Information Portal. U.S. National Library of Medicine.

Monoclonals for bone, musculoskeletal, circulatory, and neurologic systems

Bone

Human	Burosumab Denosumab
Humanized	Blosozumab^† Romosozumab

Musculoskeletal

Human	Stamulumab^†

Circulatory

Human	Abelacimab Alirocumab Ascrinvacumab^§ Bentracimab^† Enoticumab Evinacumab Evolocumab Icrucumab Inclacumab Nesvacumab Orticumab Ramucirumab Rinucumab
Mouse	Biciromab^‡ Imciromab^‡
Chimeric	Abciximab Volociximab
Humanized	Alacizumab pegol Bevacizumab/Ranibizumab Bococizumab Brolucizumab Caplacizumab Demcizumab Emicizumab Etaracizumab Faricimab Idarucizumab Ralpancizumab Tadocizumab Vanucizumab

Neurologic

Human	Anti-amyloid drugs Aducanumab Donanemab Gantenerumab^† Lecanemab Erenumab Fasinumab^† Fulranumab Opicinumab
Humanized	Bapineuzumab^† Crenezumab^† Eptinezumab Fremanezumab Galcanezumab Ozanezumab Ponezumab Refanezumab Semorinemab Solanezumab Tanezumab^†