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EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/660642/2015

EMEA/H/C/003964

EPAR summary for the public

Elocta

efmoroctocog alfa

This is a summary of the European public assessment report (EPAR) for Elocta. It explains how the Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Elocta.

For practical information about using Elocta, patients should read the package leaflet or contact their doctor or pharmacist.

What is Elocta and what is it used for?

Elocta is a medicine used to treat and prevent bleeding in patients with haemophilia A (an inherited bleeding disorder caused by lack of factor VIII). It contains the active substance efmoroctocog alfa.

How is Elocta used?

Elocta is available as a powder and solvent used to make a solution for injection. The injection is given into a vein over several minutes. The dose and frequency of the injections depend on whether Elocta is used to treat or prevent bleeding, the severity of the patient's factor VIII deficiency, the extent and location of the bleeding and the patient's condition and bodyweight.

Elocta can only be obtained with a prescription and treatment should be started under the supervision of a doctor who has experience in the treatment of haemophilia. For further information, see the summary of product characteristics (also part of the EPAR).

How does Elocta work?

Patients with haemophilia A lack factor VIII, a protein needed for normal clotting of the blood, and as a result, they bleed readily. The active substance in Elocta, efmoroctocog alfa, works in the body in the

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same way as human factor VIII. It replaces the missing factor VIII, thereby helping the blood to clot and giving temporary control of bleeding.

What benefits of Elocta have been shown in studies?

Two main studies of Elocta showed that the medicine is effective at both preventing and treating bleeding episodes.

In a study of 165 adult patients with haemophilia A, patients who were given Elocta as a tailored preventive treatment had around 3 bleeding episodes a year, which compares with 37 episodes a year in patients not given preventive treatment. In addition, when bleeding did occur, Elocta treatment was rated as 'excellent' or 'good' in more than 78% of cases, with 87% of bleeding episodes resolving with only one injection.

In a study in 69 children, Elocta was similarly effective: 2 bleeding episodes occurred per year on average and 81% of bleeding episodes resolved with only one injection.

What are the risks associated with Elocta?

Hypersensitivity (allergic) reactions are seen rarely with Elocta and these include: swelling of the face, rash, hives, tightness of the chest and difficulty breathing, burning and stinging at the injection site, chills, flushing, itching over the whole body, headache, low blood pressure, lethargy, nausea, restlessness, and a fast heartbeat. In some cases these reactions can become severe.

There is also a risk with factor VIII medicines that some patients will develop inhibitors (antibodies) against factor VIII, causing the medicine to stop working and resulting in a loss of bleeding control.

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

Why is Elocta approved?

Studies show that Elocta is effective at preventing and treating bleeding episodes in patients with haemophilia A and its safety is in line with what is expected for medicines of its kind. The Agency's Committee for Medicinal Products for Human Use (CHMP) therefore concluded that its benefits are greater than its risks and recommended that it be approved in the EU.

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

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 included in the summary of product characteristics and the package leaflet for Elocta, including the appropriate precautions to be followed by healthcare professionals and patients.

Further information can be found in the summary of the risk management plan.

Other information about Elocta

The European Commission granted a marketing authorisation valid throughout the European Union for Elocta on 19 November 2015.

The full EPAR and risk management plan summary for Elocta can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/European public assessment reports. For more

Elocta

EMA/660642/2015    Page 2/3 information about treatment with Elocta, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

The summary of the opinion of the Committee for Orphan Medicinal Products for Elocta can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation.

This summary was last updated in 11-2015.

Elocta

EMA/660642/2015