Hemlibra

Hemlibra can only be obtained with a prescription and treatment should be started by a doctor experienced in the treatment of haemophilia or bleeding disorders.

Hemlibra is available as a solution for injection under the skin in the belly, thigh or upper arm. Patients or their carers may be able to inject Hemlibra at home once they have been trained appropriately. The arm injection should only be given by a caregiver or healthcare professional.

The day before starting Hemlibra treatment, patients should stop treatment with bypassing agents (medicines used to prevent bleeding in patients with factor VIII inhibitors, such as activated prothrombin complex concentrate or recombinant factor VIIa).

The dose of Hemlibra depends on the patient’s bodyweight. The recommended dose is 3 mg per kg of bodyweight once every week for the first 4 weeks. Patients can then continue with either 1.5 mg/kg once every week, 3 mg/kg every 2 weeks, or 6 mg/kg every 4 weeks. Hemlibra is intended for long-term use.

For more information about using Hemlibra, see the package leaflet or contact your doctor or pharmacist.

Patients with haemophilia A lack factor VIII, a substance in the body that helps the blood to clot. The active substance in Hemlibra, emicizumab, is a monoclonal antibody which has been designed to do the work that factor VIII normally does – bringing together 2 clotting factors (IXa and X) as part of a chain of reactions needed for blood to clot.

Because emicizumab has a different structure to factor VIII, it is not affected by factor VIII inhibitors.

A study in 109 patients showed that Hemlibra is effective at preventing bleeding in patients with haemophilia A of any severity who have factor VIII inhibitors: patients given Hemlibra for prevention had fewer bleeds that needed to be treated (equivalent to 3 per year) than patients who received no preventive treatment (equivalent to 23 per year).

The study also enrolled patients who were already taking preventive treatment with medicines known as bypassing agents. When these patients were switched to Hemlibra, the number of treated bleeds per patient fell from the equivalent of around 16 bleeds per year before the switch to the equivalent of around 3 bleeds per year afterwards. Patients receiving Hemlibra also had better quality of life scores than those who were not given Hemlibra.

A study in 152 patients showed that Hemlibra is also effective at preventing bleeding in patients with severe haemophilia A without factor VIII inhibitors: patients given Hemlibra for prevention had around 1 bleed per year that needed to be treated, compared with 38 bleeds per year in patients who received no preventive treatment.

The most common side effects with Hemlibra (which may affect 1 in 10 people or more) are redness, itching or pain at the place where it is injected, headache and joint pain.

The most serious side effect is thrombotic microangiopathy (clots in small blood vessels, which may affect up to 1 in 100 people). Abnormal clotting, affecting between 1 and 10 in 1,000 people, may also occur and includes cavernous sinus thrombosis (clotting at the base of the brain) and superficial vein thrombosis (clotting in veins under the skin, usually in the arms or legs) with skin damage.

For the full list of side effects and restrictions with Hemlibra, see the package leaflet.

Only a few medicines, called bypassing agents, are suitable for patients with haemophilia A who have developed factor VIII inhibitors. Hemlibra reduces bleeding episodes in these patients and increases their quality of life.

Hemlibra also reduces bleeding in patients with severe haemophilia A without factor VIII inhibitors.

The side effects with Hemlibra are tolerable and information on how to manage the risks of serious side effects is in the prescribing information and educational materials.

The European Medicines Agency therefore decided that Hemlibra’s benefits are greater than its risks and it can be authorised for use in the EU.

The company that markets Hemlibra will issue educational materials for healthcare professionals, patients, carers and laboratory professionals about abnormal clotting side effects, the risk of taking Hemlibra at the same time as bypassing agents and how laboratory tests should be carried out for these patients. Materials will include product information, guides, and a patient alert card.

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Hemlibra have also been included in the summary of product characteristics and the package leaflet.

As for all medicines, data on the use of Hemlibra is continuously monitored. Side effects reported with Hemlibra are carefully evaluated and any necessary action taken to protect patients.

Hemlibra received a marketing authorisation valid throughout the EU on 23 February 2018.