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Emicizumab

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Information about Emicizumab

Emicizumab is a humanized bispecific monoclonal antibody to two human coagulation factors – factor IXa and factor X – that mimics the activity of factor VIII and is used to prevent bleeding episodes in patients with severe hemophilia A (which is caused by factor VIII deficiency).

Liver safety of Emicizumab

Emicizumab has not been linked to serum enzyme elevations during therapy or to instances of idiosyncratic acute liver injury.

Mechanism of action of Emicizumab

Emicizumab (e" mi ciz' ue mab) is a recombinant, humanized, bispecific monoclonal antibody to two human coagulation factors – factor IXa and factor X – that is used to prevent bleeding episodes in patients with severe hemophilia A. Hemophilia A is a serious genetic disease associated with deficiency in product of factor VIII leading to recurrent episodes of bleeding that result in major disability and premature death. Hemophilia A is typically treated with infusions of factor VIII isolated and concentrated from human plasma or produced by recombinant techniques. Some patients given factor VIII infusions, however, develop neutralizing antibodies which inhibit the anticoagulation activity of the infusions. Emicizumab is a recombinant bispecific monoclonal antibody that binds to both factor IXa and X, which mimics the co-factor function of factor VIII, bypassing its need in the coagulation cascade.

Clinical use of Emicizumab

In clinical trials, emicizumab was found to reduce bleeding episodes in patients with hemophilia A and antibodies to factor VIII.

FDA approval information for Emicizumab

Emicizumab was approved for use for this indication in the United States in 2017.

Dosage and administration for Emicizumab

Emicizumab is available as a solution in single dose vials of 30, 60, 105 and 150 mg under the commercial name Hemlibra. The recommended dose is 3 mg/kg by subcutaneous injection once weekly for 4 weeks, followed by 1.5 mg/kg weekly.

Side effects of Emicizumab

Side effects are not common, but can include headache, arthralgia and infection site reactions. Rare, but potentially severe adverse reactions may include hypersensitivity reactions and thromboembolic events.

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cystic fibrosis agents

enzyme replacement therapy

glucosylceramide synthase inhibitors (substrate restriction therapy)

lysosomal acid lipase deficiency agents

miscellaneous

homocystinuria agents

Huntington disease agents

Monoclonal Antibodies

Tyrosinemia Agents

Urea Cycle Disorder Agents

Hematologic Agents

Learn more about Emicizumab

Latest research (Pubmed)

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