Emicizumab

Emicizumab (trade name Hemlibra) is a humanized bispecific antibody for the treatment of haemophilia A, developed by Chugai (a subsidiary of Roche).[1] A Phase I clinical trial found that it was well tolerated by healthy subjects.[2]

Emicizumab
Monoclonal antibody
TypeWhole antibody
SourceHumanized
TargetActivated factor IX, factor X
Clinical data
Trade namesHemlibra
Other namesACE910
Routes of
administration
Subcutaneous injection
ATC code
Legal status
Legal status
  • In general: ℞ (Prescription only)
Identifiers
CAS Number
ChemSpider
  • none
UNII
KEGG
Chemical and physical data
FormulaC6434H9940N1724O2047S45
Molar mass145639.02 g·mol−1

In November 2017, it was approved in the United States for treatment of haemophilia A in those who had developed resistance to other treatments.[3] 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.[4]

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.[1][5]

References

  1. H. Spreitzer (4 July 2016). "Neue Wirkstoffe - Emicizumab". Österreichische Apothekerzeitung (in German) (14/2016).
  2. Uchida, N; Sambe, T; Yoneyama, K; Fukazawa, N; Kawanishi, T; Kobayashi, S; Shima, M (2016). "A first-in-human phase 1 study of ACE910, a novel factor VIII–mimetic bispecific antibody, in healthy subjects". Blood. 127 (13): 1633–1641. doi:10.1182/blood-2015-06-650226. PMC 4817308. PMID 26626991.
  3. "Roche hemophilia drug wins FDA nod, with a warning". Reuters. 17 November 2017.
  4. "FDA Grants Roche Breakthrough Therapy Designation on Hemophilia Drug". BioPharm International. UBM. 19 April 2018. Retrieved 20 April 2018.
  5. Shima, M; Hanabusa, H; Taki, M; Matsushita, T; Sato, T; Fukutake, K; Fukazawa, N; Yoneyama, K; Yoshida, H; Nogami, K (2016). "Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A". New England Journal of Medicine. 374 (21): 2044–53. doi:10.1056/NEJMoa1511769. PMID 27223146.
  • "Emicizumab". Drug Information Portal. U.S. National Library of Medicine.
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