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EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/320557/2014

EMEA/H/C/002813

EPAR summary for the public

Nuwiq

simoctocog alfa

This is a summary of the European public assessment report (EPAR) for Nuwiq. 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 Nuwiq.

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

What is Nuwiq and what is it used for?

Nuwiq is a medicine that contains the active substance simoctocog alfa. It is used for the treatment and prevention of bleeding in patients with haemophilia A (an inherited bleeding disorder caused by lack of factor VIII).

How is Nuwiq used?

Nuwiq 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.

Nuwiq is available as a powder and solvent that are mixed together to make a solution for injection into a vein. The dose and frequency of treatment depend on whether it is used to treat or prevent bleeding, as well as on the seriousness of the haemophilia, the extent and location of the bleeding and the patient's condition and bodyweight. For further information, see the summary of product characteristics (also part of the EPAR).

Patients or their carers may be able to administer Nuwiq themselves at home once they have been trained appropriately. For full details, see the package leaflet.

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How does Nuwiq work?

Patients with haemophilia A lack factor VIII, a protein needed for normal clotting of the blood, and this causes blood clotting problems, such as bleeding in the joints, muscles or internal organs. The active substance in Nuwiq, simoctocog alfa, works in the body in the same way as human factor VIII. It replaces the missing factor VIII, thereby helping the blood to clot and giving temporary control of the bleeding disorder.

Simoctocog alfa is made by a method known as 'recombinant DNA technology': it is made by cells into which a gene (part of the DNA) has been introduced that make them able to produce it.

What benefits of Nuwiq have been shown in studies?

Nuwiq has been shown to be effective at preventing and treating bleeding episodes in three main studies involving 113 patients with haemophilia A.

The first study involved 22 patients aged 12 years and above, who were given Nuwiq for the treatment of bleeding episodes or to prevent bleeding during surgery. A total of 986 bleeding episodes were recorded, the majority of which resolved with one injection of Nuwiq. The main measure of effectiveness was based on patients' assessment of how well treatment worked. Treatment with Nuwiq was rated as 'excellent' or 'good' for 94% of bleeding episodes. In the two surgeries that occurred during the study, Nuwiq was rated as excellent in preventing bleeding episodes.

The second study involved 32 patients aged 12 years and above who were given Nuwiq to prevent and treat bleeding events as well as to prevent bleeding during surgery. When used to prevent bleeding, an average of 0.19 bleeds per month was recorded for each patient. When used for the treatment of bleeding episodes, Nuwiq was mainly rated as 'excellent' or 'good' at treating major bleeding episodes and most bleeding episodes resolved following one or more injections of Nuwiq. In the five surgeries that occurred during the study, Nuwiq was rated as excellent in preventing bleeding episodes for four surgeries and as moderate to prevent bleeding during one surgical intervention.

The third study involved 59 children aged two to 12 years. When Nuwiq was used to prevent bleeding, an average of 0.34 bleeds per month was recorded for each child. When used for treatment bleeding episodes resolved in 81% of cases following one or two injections with Nuwiq.

What are the risks associated with Nuwiq?

Side effects with Nuwiq have not been commonly reported (affecting between 1 and 10 in 1,000 people). They include paraesthesia (unusual sensations like pins and needles), headache, vertigo (a spinning sensation), dry mouth, back pain and inflammation and pain at the injection site.

Hypersensitivity (allergic) reactions although not seen so far with Nuwiq, have been reported rarely with FVIII products and may in some cases progress to severe allergic reactions. Following treatment with FVIII products some patients may develop factor VIII inhibitors, which are antibodies (proteins) that the body's immune system produces against factor VIII and which can cause the medicine to stop working, resulting in a loss of bleeding control. In such cases, a specialised haemophilia centre should be contacted.

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

Why is Nuwiq approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Nuwiq's benefits are greater than its risks and recommended that it be approved for use in the EU. The CHMP concluded that Nuwiq has been shown to be effective at treating and preventing the occurrence of bleeding in patients with haemophilia A. Nuwiq was also effective in preventing and treating bleeding in relation to surgery bleeding episodes, with effects similar to other factor VIII products. The safety profile of Nuwiq was also considered similar to other factor VIII products.

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

A risk management plan has been developed to ensure that Nuwiq 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 Nuwiq, 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 Nuwiq

The European Commission granted a marketing authorisation valid throughout the European Union for Nuwiq on 24 July 2014.

The full EPAR and risk management plan summary for Nuwiq can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/European public assessment reports. For more information about treatment with Nuwiq, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

This summary was last updated in 07-2014.

Nuwiq

EMA/320557/2014

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