Iblias - Octocog Alfa

What is Iblias - Octocog Alfa and what is it used for?

Iblias is a medicine used to treat and prevent bleeding in patients with haemophilia A (an inherited bleeding disorder caused by the lack of a coagulation protein called factor VIII). It contains the active ingredient octocog alfa, equal to the human factor VIII.

How is Iblias - Octocog Alfa used?

Iblias is available as a powder and solvent used to make a solution for injection. Administration by injection into a vein requires several minutes. The dose and duration of therapy depend on whether Iblias is used as a preventive or curing of bleeding, on the severity of the haemophilia, on the extent and location of the bleeding, and on the patient's health and body weight.

Iblias can only be obtained with a prescription and treatment must be performed under the supervision of a doctor experienced in treating haemophilia. For more information, see the summary of product characteristics (included with EPAR).

How does Iblias - Octocog Alfa work?

Patients with haemophilia A lack factor VIII, a protein necessary for blood clotting; consequently, they are easily prone to bleeding and may have problems such as bleeding from joints, muscles or internal organs. The active substance in Iblias, octocog alfa, works in the body in the same way as the human factor VIII: it replaces the missing factor VIII, favoring blood coagulation and allowing temporary control of the bleeding disorder.

What benefits of Iblias - Octocog Alfa have been shown in studies?

The efficacy of Iblias in the prevention and treatment of bleeding has been demonstrated in one main study involving 62 patients aged 12 years or older with severe haemophilia A and previously treated with other factor VIII products. The number of bleedings observed during Iblias therapy was, on average, 3.8 events per year (mainly in the joints). Prior to Iblias therapy, bleeding events were, on average, 6.9 per year. Comparable results were observed in patients who continued to take the medicine after the initial study was completed.

About 70% of the bleeding events occurred were managed with a single Iblias injection, a further 15% of the events responded to a second injection and the response was considered good or excellent in about 80% of cases. Furthermore, the control of blood loss observed in 12 patients who underwent major surgery during the study was rated good or excellent by the doctors of these patients.

A second study was conducted on 51 children aged less than 12 years, previously treated with other factor VIII-based products, for which the same result of 3.8 bleeding events per year was found, on average, during therapy with Iblias (especially in relation to trauma). Treatment response was considered good or excellent in about 90% of cases.

Data from a supportive study also confirmed the benefits of preventive treatment with Iblias in reducing the number of bleedings.

What are the risks associated with Iblias - Octocog Alfa?

Hypersensitivity (allergic) reactions with Iblias are uncommon, affecting from 1 in 1, 000 to less than 1 in 100 patients. When they occur they may include: angioedema (swelling of subcutaneous tissues), burning and stinging at the injection site, chills, redness, itchy rash, headache, hives, hypotension (low blood pressure), lethargy, nausea, restlessness, tachycardia (rapid heart beat), chest constriction, tingling, vomiting and respiratory wheezing. In some cases, these reactions can occur in severe form.

Patients may develop antibodies against hamster or mouse proteins; the medicine must not be used in patients who are hypersensitive (allergic) to octocog alfa or to hamster or mouse proteins. With factor VIII medicines, there is also a risk that some patients develop inhibitors (antibodies) against this factor; therefore, the medicine may become ineffective, resulting in loss of control over bleeding.

For the full list of limitations and side effects reported with Iblias, see the package leaflet.

Why was Iblias - Octocog Alfa approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Iblias's benefits are greater than its risks and recommended that it be approved for use in the EU. Iblias has been shown to be effective in both prevention and treatment of bleeding, including the management of blood loss during surgery, and can be used in all age groups. Regarding safety, the effects reported are in line with those expected for a factor VIII based product. Ongoing studies should provide additional evidence on efficacy and safety in patients not previously treated with factor VIII-based medicines as well as additional data on long-term use in children.

What measures are being taken to ensure the safe and effective use of Iblias - Octocog Alfa?

A risk management plan has been developed to ensure that Iblias is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the Iblias package leaflet, including the appropriate precautions to be followed by healthcare professionals and patients.

Further information is available in the summary of the risk management plan.

Furthermore, the company that markets Iblias will carry out studies aimed at assessing the safety and efficacy of the medicine in patients who have not already been treated with other products based on factor VIII and to provide further evidence on the safety and effectiveness of the treatment long term with Iblias in children.