Kovaltry - Octocog Alfa

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

Kovaltry 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 Kovaltry used - Octocog Alfa?

Kovaltry is available in powder and solvent form, used for the preparation of a solution for injection. Administration by injection into a vein requires several minutes. The dose and frequency of injections depend on whether Kovaltry is used as a preventive or curing of bleeding, on the severity of the factor VIII deficiency, on the extent and location of the bleeding, and on the health conditions and body weight of the patient.

Kovaltry 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 Kovaltry - Octocog Alfa work?

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

What benefits of Kovaltry - Octocog Alpha have been shown in studies?

The effectiveness of Kovaltry in the prevention and treatment of bleeding has been demonstrated in a main study conducted on 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 Kovaltry therapy was, on average, 3.8 events per year (mainly in the joints). Prior to Kovaltry therapy, the bleeding events found 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. C

approximately 70% of the bleeding events occurred were managed with a single Kovaltry 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 Kovaltry therapy (especially in relation to trauma). Treatment response was considered good or excellent in about 90% of cases.

Even the data obtained from a support study confirmed the benefits of preventive treatment with Kovaltry in reducing the number of bleedings.

What are the risks associated with Kovaltry - Octocog Alfa?

Hypersensitivity (allergic) reactions with Kovaltry are uncommon, affecting 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 tightness, 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 Kovaltry, see the package leaflet.

Why has Kovaltry - Octocog Alfa been approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Kovaltry's benefits are greater than its risks and recommended that it be approved for use in the EU. Kovaltry has been shown to be effective both in prevention and in the 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 Kovaltry - Octocog Alfa?

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

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

In addition, the company that markets Kovaltry 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 efficacy of the treatment at long term with Kovaltry in children.