Hemlibra approved by the FDA
In November 2017, the U.S. Food and Drug Administration approved Hemlibra (emicizumab-kxwh) for the prevention and reduction in frequency of bleeding episodes in children and adults with hemophilia A who have inhibitors. The new drug is made by Genentech. Approval was granted based on data from two clinical trials, Haven 1 and Haven 2 (the study name is ACE910).
Hemlibra is the first drug approved that stops bleeding by bridging Factor IXa and Factor X using a monoclonal antibody. It is given weekly via injection under the skin (subcutaneous). This is quite different from the current treatment of injecting factor VIII directly into a vein. Dosing is 3mg/kg weekly for the first 4 weeks, then reduced to 1.5mg/kg weekly. Hemlibra is dosed in milligrams, unlike factor VIII which is dosed in international units. The product will be available in four different sizes: 30mg, 60mg, 105mg, and 150mg single use vials. The dosage is based on body weight. It may be stored in the refrigerator up to the expiration date printed on each vial, or at room temperature for a total of 7 days.
Patients treated with Hemlibra reported an improvement in hemophilia-related symptoms, such as painful swelling, joint pain, and difficulty walking, compared to patients who did not receive the weekly treatment. The most common side effects are injection-site reactions and headaches. There is a black-box label warning on the Hemlibra package that alerts patients and healthcare providers to a possible serious reaction. Severe blood clots and thrombotic microangiopathy (TMA) were observed in study patients who were given an activated prothrombin complex concentrate (aPCC -FEIBA®) while taking Hemlibra.
This new therapy has only been approved for hemophilia A patients with inhibitors. If you are interested in learning more about Hemlibra, please read the patient information on the Hemlibra website and contact your Hemophilia Treatment Center. The Hemophilia of Georgia and Beacon pharmacies are working with commercial and government payers to gain coverage for clients. We will be glad to answer any questions you may have.