Emicizumab

type	mab
mab type	mab
source	zu
target	Activated factor IX, factor X
tradename	Hemlibra
medlineplus	a622046
dailymedid	Emicizumab
pregnancy au	B2
routes of administration	Subcutaneous
atc prefix	B02
atc suffix	BX06
legal au	S4
legal au comment	‡R1R‡
legal ca	Rx-only
legal ca comment	/ Schedule D‡R2R‡
legal us	Rx-only
legal us comment	‡R3R‡
legal eu	Rx-only
legal status	Rx-only
cas number	1610943-06-0
drugbank	DB13923
chemspiderid	none
unii	7NL2E3F6K3
kegg	D10821
synonyms	ACE910, RG6013, emicizumab-kxwh
c	6434
h	9940
n	1724
o	2047
s	45

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][5][6]

Emicizumab was first approved by the U.S. Food and Drug Administration (FDA) in November 2017 for routine prophylaxis in patients with hemophilia A who have developed factor VIII inhibitors.[7][8]

Research

A Phase I clinical trial found that it was well tolerated by healthy subjects.[18]

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.[19]

HAVEN clinical trial program

Emicizumab was evaluated in one of the largest pivotal clinical trial programs in hemophilia A, comprising four pivotal Phase III studies known as the HAVEN trials.[20]

HAVEN 1 study

HAVEN 1 was a randomized, multicenter, open-label Phase III trial that included 109 male patients (ages 12–75 years) with severe hemophilia A and factor VIII inhibitors.[21] The study demonstrated that emicizumab prophylaxis significantly reduced bleeding episodes compared to no prophylaxis in patients with inhibitors.[22][23][24]

HAVEN 3 study

HAVEN 3 evaluated emicizumab in patients with severe hemophilia A without factor VIII inhibitors. The trial demonstrated that subcutaneous emicizumab prophylaxis, administered either once weekly or every two weeks, resulted in bleeding rates that were significantly lower by more than 95% compared to no prophylaxis.[6] More than 55% of participants in each randomized emicizumab regimen experienced no treated bleeds during the study period.[6][25]

HAVEN 1 study

HAVEN 3 study

Additional HAVEN studies

The clinical program also included HAVEN 2 (pediatric patients with inhibitors) and HAVEN 4 (patients switching from bypassing agent prophylaxis), further establishing the efficacy and safety profile of emicizumab across different patient populations.[26]

Emicizumab

Medical uses

Available forms

Adverse effects

Pharmacology

Pharmacokinetics

Mechanism of action

Research

HAVEN clinical trial program

HAVEN 1 study

HAVEN 3 study

HAVEN 1 study

HAVEN 3 study

Additional HAVEN studies

Regulatory approvals

See also

References