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EUROPEAN MEDICINES AGENCY

SCIENCE MEDICINES HEALTH

EMA/75512/2016

EMEA/H/C/003855

EPAR summary for the public

Coagadex

human coagulation factor X

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

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

What is Coagadex and what is it used for?

Coagadex is a medicine used for the treatment and prevention of bleeding (including during and after an operation) in patients with hereditary factor X deficiency. Factor X deficiency is a bleeding disorder caused by lack of factor X, a protein needed for normal clotting of the blood.

Because the number of patients with factor X deficiency is low, the disease is considered 'rare', and Coagadex was designated an 'orphan medicine' (a medicine used in rare diseases) on 14 September 2007.

Coagadex contains the active substance human coagulation factor X.

How is Coagadex used?

Coagadex is available as a powder and solvent used to make a solution for injection into a vein. The dose and frequency of the injections depend on the severity of the patient's factor X deficiency, the extent and location of the bleeding and the patient's condition and bodyweight.

Coagadex can only be obtained with a prescription and treatment should be started under the supervision of a doctor experienced in the treatment of rare bleeding disorders. Patients may inject Coagadex themselves at home once they have been trained appropriately. For further information, see the package leaflet.

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© European Medicines Agency, 2016. Reproduction is authorised provided the source is acknowledged.

How does Coagadex work?

Patients with hereditary factor X deficiency lack factor X, a protein needed to form the scab (blood clot) that stops wounds from bleeding. In these patients, blood clots do not form properly, resulting in longer bleeding time and poor wound healing. Blood may seep into surrounding tissues, resulting in local pain and swelling. Bleeding may also occur in internal organs. The active substance in Coagadex is human factor X isolated from the plasma of blood donors. It replaces the missing factor X, thereby helping the blood to clot and giving temporary control of bleeding.

What benefits of Coagadex have been shown in studies?

Coagadex has been investigated in one main study involving 16 patients with factor X deficiency aged 12 to 42 years. Patients in the study received Coagadex to treat any spontaneous bleeding that occurred during the treatment period or to prevent bleeding during surgery. The main measure of effectiveness was based on the doctor's and patient's assessment of how well the treatment worked in preventing and treating bleeding episodes.

For the treatment of bleeding, 187 bleeding episodes were recorded and assessed, and treatment with Coagadex was rated as 'excellent' or 'good' for 98.4% of bleeding episodes. In the three minor surgeries that occurred during the study, Coagadex was rated as excellent in preventing bleeding episodes.

What are the risks associated with Coagadex?

The most common side effects with Coagadex (which may affect up to 1 in 10 people) are pain or redness at the injection site, fatigue (tiredness), and back pain.

Hypersensitivity (allergic) reactions have been observed rarely in patients treated for bleeding disorders (in up to 1 patient in 1,000), and may include: angioedema (swelling of tissues under the skin), burning and stinging at the injection site, chills, flushing, itchy rash over the whole body, headache, hives, hypotension (low blood pressure), lethargy, nausea (feeling sick), restlessness, tachycardia (rapid heartbeat), tightness of the chest, tingling, vomiting and wheezing. These reactions have not been reported during clinical studies with Coagadex.

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

Why is Coagadex approved?

The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that the benefits of Coagadex are greater than its risks and recommended that it be approved for use in the EU. The CHMP considered the lack of specific therapies for factor X deficiency, and concluded that Coagadex has been shown to be effective at treating and preventing the occurrence of bleeding in patients above 12 years with the condition. Preliminary data in children below 12 are consistent with those obtained in adolescents up to 17 years of age. The safety profile of Coagadex was considered satisfactory based on available data, with side effects that were manageable and of mild or moderate intensity. However given the extreme rarity of the condition, the safety database is small and rare events are not expected to be captured during the clinical studies.

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

A risk management plan has been developed to ensure that Coagadex 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 Coagadex, including the appropriate precautions to be followed by healthcare professionals and patients.

Other information about Coagadex

The European Commission granted a marketing authorisation valid throughout the European Union for Coagadex on 16 March 2016.

The full EPAR for Coagadex 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 Coagadex, 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 Coagadex can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation.

This summary was last updated in 03-2016.

Coagadex

EMA/75512/2016

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