What is Idelvion - Albutrepenonacog alfa and what is it used for?
Idelvion is a medicine used to prevent and treat bleeding in patients with haemophilia B, a hereditary bleeding disorder caused by the lack of a coagulation protein called factor IX. It can be given to patients of any age.
Because the number of patients with haemophilia B is low, the disease is considered 'rare' and Idelvion was designated an 'orphan medicine' (a medicine used in rare diseases) on 4 February 2010.
It contains the active ingredient albutrepenonacog alfa.
How is Idelvion - Albutrepenonacog alfa used?
Idelvion can only be obtained with a prescription and treatment must be carried out under the supervision of a doctor experienced in treating haemophilia.
Idelvion is available as a powder and solvent, which when mixed together form a solution for injection into a vein. The dose and frequency of injections depend on the patient's body weight and whether Idelvion is used for preventive or curing bleeding, the severity of the patient's factor IX deficiency, the extent and location of the bleeding and the health conditions and age of the patient. For more information on the use of the medicine, see the summary of product characteristics (also part of the EPAR).
How does Idelvion - Albutrepenonacog alfa work?
Patients with haemophilia B are deficient in factor IX, a protein necessary for normal blood coagulation, and, as a result, are easily prone to bleeding. The active substance in Idelvion, albutrepenonacog alfa, works in the body in the same way as the human factor IX. Replaces the missing factor IX, promoting blood coagulation and allowing temporary bleeding control.
What benefit has Idelvion - Albutrepenonacog alfa shown during the studies?
In a study of 80 adult and adolescent patients and in another study involving 27 children under the age of 12, Idelvion was effective in preventing bleeding and most patients had no bleeding during preventive treatment . Furthermore, Idelvion has been shown to be effective in treating bleeding episodes when they occurred; approximately 93% of bleeding episodes were resolved with a single injection of Idelvion.
What is the risk associated with Idelvion - Albutrepenonacog alfa?
Hypersensitivity (allergic) reactions to Idelvion have been reported rarely and include: swelling, burning and stinging at the injection site, chills, redness, rash, headache, hives, low blood pressure, lethargy, nausea and vomiting, agitation, tachycardia, chest tightness and wheezing. In some cases, these reactions can occur in severe form.
With factor IX medicines there is also the risk that some patients develop inhibitors (antibodies) against this factor; therefore, the medicine may become ineffective, resulting in loss of control over bleeding. Factor IX medicines can also cause problems due to the formation of blood clots in the blood vessels. For the full list of all side effects reported with Idelvion, see the package leaflet.
Idelvion should not be used in patients who are hypersensitive (allergic) to the active substance or to any of the other ingredients of the medicine. It must also not be given to patients who are allergic to hamster proteins.
Why has Idelvion - Albutrepenonacog alfa been approved?
Studies show that Idelvion is effective in preventing and treating bleeding episodes in patients with haemophilia B and that its safety profile is comparable to that of other factor IX products. The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Idelvion's benefits are greater than its risks and recommended that it be approved for use in the EU.
What measures are being taken to ensure the safe and effective use of Idelvion - Albutrepenonacog alfa?
A risk management plan has been developed to ensure that Idelvion 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 Idelvion, including the appropriate precautions to be followed by healthcare professionals and patients.
More information on Idelvion - Albutrepenonacog alfa
For the full LONSurf EPAR consult the Agency website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about Lonsurf therapy, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
