Elocta - Efmoroctocog alfa

What is Elocta - Efmoroctocog alfa and what is it used for?

Elocta is used for the treatment and prevention of bleeding in patients with haemophilia A (an inherited bleeding disorder caused by factor VIII deficiency). The active substance contained in it is efmoroctocog alfa.

How is Elocta - Efmoroctocog alfa used?

Elocta is available as a powder and solvent that can be used to make a solution for injection. Administration by injection into a vein requires several minutes. The dose and duration of therapy depend on the preventive or curative use of the medicine, on the severity of the haemophilia, on the extent and location of the bleeding, as well as on the patient's health and body weight.

Elocta is available only on prescription and treatment should be started under the supervision of a doctor experienced in treating haemophilia. For more information refer to the summary of product characteristics (also included with the EPAR).

How does Elocta - Efmoroctocog alfa work?

Patients with haemophilia A are deficient in factor VIII, a protein necessary for normal blood coagulation, and, therefore, are easily prone to bleeding. The active substance in Elocta, efmoroctocog alfa, works in the body in the same way as the human factor VIII. It replaces the missing factor VIII, favoring blood coagulation and ensuring a temporary control of the coagulation disorder.

What benefit has Elocta - Efmoroctocog alfa shown during the studies?

The two main studies conducted on Elocta have shown that the medicine is effective both for the prevention and treatment of bleeding episodes.

In a study of 165 adult patients with haemophilia A, patients who received Elocta as a personalized preventive treatment experienced approximately 3 bleeding episodes per year, compared to 37 episodes per year in patients not subjected to preventive treatment. Furthermore, during bleeding, treatment with Elocta was assessed as "excellent" or "good" in over 78% of cases, with 87% of bleeding episodes resolved after a single injection.

In a study of 69 children, Elocta was equally effective: on average, 2 bleeding episodes each year, and in 81% of cases the episodes resolved after a single injection.

What is the risk associated with Elocta - Efmoroctocog alfa?

Elocta has only rarely produced hypersensitivity (allergic) reactions, including: swelling of the face, rash, hives, chest tightness and difficulty breathing, burning and pain at the injection site, chills, flushing, itching all over the body, headache, low blood pressure, lethargy, nausea, restlessness and rapid heartbeat. In some cases, these reactions can occur in severe form.

There is also a risk that some patients develop inhibitors (antibodies) directed against factor VIII and therefore the medicine is no longer effective, resulting in loss of control over bleeding.

For the full list of side effects and restrictions related to Elocta, refer to the package leaflet.

Why has Elocta - Efmoroctocog alfa been approved?

Studies show that Elocta is effective in preventing and treating bleeding episodes in patients with haemophilia A and that its safety profile is in line with that expected for such drugs. The Agency's Committee for Medicinal Products for Human Use (CHMP) therefore decided that its benefits outweigh its risks and recommended its authorization for use in the EU.

What measures are being taken to ensure the safe and effective use of Elocta - Efmoroctocog alfa?

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