What is Alprolix - Eftrenonacog Alfa and what is it used for?
Alprolix is a medicine used to treat and prevent bleeding in patients with haemophilia B, an inherited 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 Alprolix was designated an 'orphan medicine' (a medicine used in rare diseases) on 8 June 2007.
Alprolix contains the active substance eftrenonacog alfa.
How is Alprolix - Eftrenonacog Alfa used?
Alprolix can only be obtained with a prescription and treatment must be supervised by a doctor experienced in treating haemophilia.
Alprolix is available as a powder and solvent which, when mixed, form an intravenous solution for injection. The dose and frequency of administration depend on the patient's body weight, whether Alprolix is used for preventive or curative purposes of bleeding, the severity of the factor IX deficiency in the patient, the extent and location of the bleeding, as well as from the health conditions and the age of the patient. For more information on the use of this medicine, see the summary of product characteristics (also part of the EPAR).
Patients, or people who care for them, can give themselves, or be given, Aplorix alone at home, after receiving appropriate instructions. For more information, read the package leaflet.
How does Alprolix - Eftrenonacog Alfa work?
Patients with haemophilia B have a factor IX deficiency, a protein necessary for normal blood coagulation. As a result, they are easily prone to bleeding. The active substance in Alprolix, eftrenonacog 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 Alprolix - Eftrenonacog Alfa shown during the studies?
Alprolix has been shown to be effective in preventing and treating bleeding events in two main studies involving patients with haemophilia B.
In the first study of 123 adults and adolescents over 12 years, 3 patients had been given Alprolix as a preventive treatment once a week and 3 bleeding episodes a year occurred; in patients who had been given Alprolix as a preventive treatment once every 10 days there were 2 bleeding episodes a year; finally, about 18 bleeding episodes a year occurred in patients who received Alrpolix to treat bleeding when needed. Furthermore, at the occurrence of bleeding, approximately 90% of the episodes were resolved with a single injection.
In the second study, conducted on 30 children under the age of 12, the efficacy of Alprolix was similar: on average 2 bleeding episodes were found per year and in 75% of cases the episodes were resolved after a single injection.
What is the risk associated with Alprolix - Eftrenonacog Alfa?
Hypersensitivity (allergic) reactions to Alprolix have been reported rarely and include: swelling, burning and stinging at the site of injection, chills, flushing, itchy rashes, headache, hives, low blood pressure, lethargy, nausea and vomiting, restlessness, 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 the factor in question; 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 restrictions and side effects reported with Alprolix, see the package leaflet.
Why has Alprolix - Eftrenonacog Alfa been approved?
Studies show that Alprolix 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 medicines. The Agency's Committee for Medicinal Products for Human Use (CHMP) therefore decided that Alprolix's benefits are greater than its risks and recommended that it be used in the EU.
What measures are being taken to ensure the safe and effective use of Alprolix - Eftrenonacog Alfa?
A risk management plan has been developed to ensure that Alprolix 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 Alprolix, including the appropriate precautions to be followed by healthcare professionals and patients.
More information on Alprolix - Eftrenonacog Alfa
For the full EPAR for Alprolix, consult the Agency's website: ema.europa.eu/Find medicine / Human medicines / European public assessment reports. For more information about treatment with Alprolix, 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 Alprolix is available on the Agency's website: ema.europa.eu/Find medicine / Human medicines / Rare disease designation.
