What is and what is Coagadex used for - Human X-coagulation factor X?
Coagadex is a medicine used to treat and prevent bleeding (even during or after surgery) in patients with an inherited factor X deficiency. Factor X deficiency is a bleeding disorder caused by the lack of factor X, a necessary protein for normal blood clotting.
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 September 14, 2007.
Coagadex contains the active ingredient called factor X of human coagulation.
How is Coagadex used - Factor X of human coagulation?
Coagadex is available in the form of a powder and solvent used for the preparation of an intravenous solution for injection. The dose and frequency of injections depend on the severity of the factor X deficiency, on the extent and location of the bleeding, and on the patient's health and body weight.
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 can practice Coagadex self-injection at home after receiving appropriate instructions. For more information, see the package leaflet.
How does Coagadex - a factor in human coagulation?
Patients suffering from hereditary factor X deficiency are characterized by the lack of factor X, a protein necessary for the formation of the crust (blood clot) that stops bleeding from the wound. In these patients, blood clots do not form properly and this causes prolonged bleeding and poor wound healing. The blood can infiltrate the surrounding tissues and cause local pain and swelling. Bleeding may also occur on the internal organs. The active substance in Coagadex is the human factor X isolated from the plasma of blood donors. Replaces the missing X factor, promoting blood coagulation and allowing temporary bleeding control.
What benefit has Coagadex - Factor X of human coagulation shown during the studies?
Coagadex has been studied in one main study involving 16 patients with factor X deficiency, aged 12 to 42 years. Patients who participated in the study received Coagadex as a therapy for spontaneous bleeding that occurred during the treatment period or to prevent bleeding during surgery. The main efficacy parameter was based on the evaluation by the doctor and the patient of the efficacy of the treatment in the prevention and treatment of bleeding episodes.
Regarding the treatment of hemorrhage, 187 episodes were recorded and examined and Coagadex treatment was rated "excellent" or "good" in 98.4% of bleeding episodes. In the three minor surgical operations performed during the study, Coagadex therapy was considered "excellent" in preventing bleeding episodes.
What is the risk associated with Coagadex - Factor X of human coagulation?
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 (up to 1 in 1, 000 patients) in patients treated for bleeding disorders and may include: angioedema (swelling of subcutaneous tissues), burning and stinging at the injection site, chills, redness, itchy rash on the whole body, headache, hives, hypotension (low blood pressure), lethargy, nausea (feeling sick), restlessness, tachycardia (rapid heart beat), chest tightness, tingling, vomiting and respiratory wheezing. These reactions were not found during clinical studies conducted on Coagadex.
For the full list of restrictions and side effects reported with Coagadex, see the package leaflet.
Why has Coagadex - Human clotting factor X been approved?
The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Coagadex's benefits are greater than its risks and recommended that it be approved for use in the EU. The CHMP, in view of the lack of specific therapies for factor X deficiency, considered that Coagadex has been shown to be effective in the treatment and prevention of bleeding episodes in patients over 12 years of age with this condition. Preliminary data for children under the age of 12 are in line with those obtained in adolescent patients up to 17 years of age. The safety profile of Coagadex was judged satisfactory based on the available data, with mild to moderate side effects that could be managed. However, due to the extreme rarity of the disease the safety database is limited and there are no plans to record rare events during clinical trials.
What measures are being taken to ensure the safe and effective use of Coagadex - Factor X of human coagulation?
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 on Coagadex - Factor X of human coagulation
For the full EPAR for Coagadex, consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information on Coagadex therapy, 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 related to Coagadex is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
