Hemlibra (emicizumab-kxwh)

The following drug information is obtained from various newswires, published medical journal articles, and medical conference presentations.

Company:

Approval Status:

Approved November 2017

Specific Treatments:

prevention or reduction of bleeding episodes in patients with hemophilia A

Therapeutic Areas

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General Information

Hemlibra (emicizumab-kxwh) is a bispecific factor IXa- and factor X-directed antibody. 

Hemlibra is specifically indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. 

Hemlibra is supplied as a solution for subcutaneous injection. The recommended dose is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly. 

Clinical Results

FDA Approval

The FDA approval of Hemlibra was based on data from two clinical trials. The first was a trial that included 109 males aged 12 and older with hemophilia A with FVIII inhibitors. The randomized portion of the trial compared Hemlibra to no prophylactic treatment in 53 patients who were previously treated with on-demand therapy with a bypassing agent before enrolling in the trial. Patients taking Hemlibra experienced approximately 2.9 treated bleeding episodes per year compared to approximately 23.3 treated bleeding episodes per year for patients who did not receive prophylactic treatment. This represents an 87 percent reduction in the rate of treated bleeds. The trial also included patient-reported Quality of Life metrics on physical health. Patients treated with Hemlibra reported an improvement in hemophilia-related symptoms (painful swellings and joint pain) and physical functioning (pain with movement and difficulty walking) compared to patients who did not receive prophylactic treatment. The second trial was a single arm trial of 23 males under the age of 12 with hemophilia A with FVIII inhibitors. During the trial, 87 percent of the patients taking Hemlibra did not experience a bleeding episode that required treatment.

Side Effects

Adverse effects associated with the use of Hemlibra may include, but are not limited to, the following:

  • injection site reactions
  • headache
  • arthralgia

The Hemlibra drug label comes with the following Boxed Warning: Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving Hemlibra prophylaxis. Monitor for the development of thrombotic microangiopathy and thrombotic events if aPCC is administered. Discontinue aPCC and suspend dosing of Hemlibra if symptoms occur. 

Mechanism of Action

Hemlibra (emicizumab-kxwh) is a bispecific factor IXa- and factor X-directed antibody. This restores the function of missing activated factor VIII that is needed for effective hemostasis.

Additional Information

For additional information regarding Hemlibra or hemophilia A, please visit https://www.hemlibra.com/