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EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/308649/2012

EMEA/H/C/000276

EPAR summary for the public

Helixate NexGen

human coagulation factor VIII (octocog alfa)

This document is a summary of the European public assessment report (EPAR) for Helixate NexGen. It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Helixate NexGen.

What is Helixate NexGen?

Helixate NexGen is a powder and solvent that are mixed together to make up a solution for injection. Helixate NexGen contains the active substance human coagulation factor VIII (octocog alfa).

What is Helixate NexGen used for?

Helixate NexGen is used for the treatment and prevention of bleeding in patients with haemophilia A (an inherited bleeding disorder). Helixate NexGen is intended for either short-term or long-term use.

The medicine can only be obtained with a prescription.

How is Helixate NexGen used?

Helixate NexGen should be started by a doctor who has experience in the treatment of haemophilia.

Helixate NexGen is given by injection into a vein lasting several minutes. The dose and frequency of injection depend on whether Helixate NexGen is used to treat or prevent bleeding, or to reduce bleeding during surgery. The dose is adjusted depending on the severity and location of the bleeding, or the type of surgery. Helixate NexGen can also be given continuously for up to seven days as an infusion (drip into a vein) in patients undergoing major surgery.

Full details on how to calculate the doses are included in the summary of product characteristics (also part of the EPAR).

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How does Helixate NexGen work?

The active substance in Helixate NexGen, human coagulation factor VIII, is a substance that helps the blood to clot. Patients with haemophilia A lack factor VIII, which causes blood clotting problems such as bleeding in the joints, muscles and internal organs. Helixate NexGen is used to correct the factor VIII deficiency by replacing the missing factor VIII, giving temporary control of the bleeding disorder.

The human coagulation factor VIII in Helixate NexGen is not extracted from human blood but is produced by a method known as 'recombinant DNA technology': it is made by a cell that has received a gene (DNA), which makes it able to produce human coagulation factor VIII.

How has Helixate NexGen been studied?

Helixate NexGen is similar to another medicine that was previously authorised in the European Union (EU) called Helixate, but it is prepared differently so that there are no human-derived proteins in the medicine. Because of this, Helixate NexGen has been compared with Helixate to show that the two medicines are equivalent.

Helixate NexGen given as an injection into a vein has been studied in 66 patients who had previously been treated with recombinant human coagulation factor VIII and 61 children who had not. The main measure of effectiveness in the studies was the number of treatments required to stop each new bleed.

Helixate NexGen as a continuous infusion has also been studied in 15 patients with haemophilia A undergoing major surgery. The main measure of effectiveness was the doctor's assessment of how well bleeding was stopped.

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. Helixate NexGen given at high dose has been studied to see whether it is effective in clearing antibodies against factor VIII from the blood (a process known as immune tolerance induction) so that treatment with factor VIII remains effective.

What benefit has Helixate NexGen shown during the studies?

In the previously treated patients, overall, 95% of bleeds responded to one or two injections of Helixate NexGen into a vein. In the previously untreated patients, about 90% of bleeding episodes responded to treatment with one or two injections into a vein.

When given as a continuous infusion, stopping bleeding was assessed as 'excellent' in all 15 patients.

The data provided on immune tolerance induction in patients with inhibitors showed, that some patients benefit from the high dosage and the inhibitor could be eliminated, nevertheless the data were not considered sufficient to specifically approve the medicine for this use.

What is the risk associated with Helixate NexGen?

The most common side effects with Helixate NexGen (seen in between 1 and 10 patients in 100) include the development of antibodies against factor VIII, mainly in patients who have previously not been treated with factor VIII, reactions at the infusion site and skin-associated hypersensitivity (allergic) reactions (itching, hives and rash). For the full list of all side effects reported with Helixate NexGen, see the package leaflet.

Helixate NexGen must not be used in people who are known to be hypersensitive (allergic) to human coagulation factor VIII or to any of the other ingredients. It must also not be used in people who are known to have allergic reactions to mouse or hamster protein.

Why has Helixate NexGen been approved?

The CHMP decided that Helixate NexGen's benefits are greater than its risks and recommended that it be given marketing authorisation.

Other information about Helixate NexGen:

The European Commission granted a marketing authorisation valid throughout the EU for Helixate NexGen on 4 August 2000.

The full EPAR for Helixate NexGen 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 Helixate NexGen, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

This summary was last updated in 05-2012.

Helixate NexGen