What is Rixubis - what is nonacog gamma and what is it used for?
Rixubis is a medicine used to treat and prevent bleeding in patients with haemophilia B, an inherited coagulation disorder caused by a lack of factor IX. It can be used in patients of all ages, for short or long term treatment. Rixubis contains the nonacog gamma active ingredient.
How is Rixubis used - nonacog gamma?
Rixubis can only be obtained with a prescription and treatment should be started under the supervision of a doctor experienced in treating haemophilia. Rixubis is available as a powder and solvent, which are mixed to produce a solution to be injected into a vein. The dose and frequency of treatment depend on the patient's body weight and whether Rixubis is used for the treatment or prevention of bleeding, as well as the severity of the haemophilia, the extent and location of the bleeding and age and from the patient's state of health. For more information, see the summary of product characteristics (included with EPAR). Patients or caregivers can administer Rixubis at home once they have received appropriate training. For more information, see the package leaflet.
How does Rixubis work - nonacog range?
Patients with haemophilia B have a lack of factor IX, which is necessary for blood to clot properly. This deficiency causes clotting problems, such as bleeding in the joints, muscles or internal organs. The active substance in Rixubis, nonacog gamma, is a version of human factor IX and helps the blood to coagulate in the same way. Rixubis can therefore be used to replace the missing factor IX, allowing temporary control of the coagulation disorder. Nonacog gamma is not extracted from human blood, but is produced by a method known as "recombinant DNA technology": it is made from hamster cells in which a gene (DNA) has been introduced, which allows cells to produce the factor of human coagulation.
What benefit has Rixubis shown - nonacog gamma during the studies?
The benefits of Rixubis in the treatment and prevention of bleeding episodes have been demonstrated in three main studies conducted in patients with severe or moderately severe haemophilia B. None of the studies compared the efficacy of Rixubis directly with another medicine. Efficacy in stopping bleeding was measured on a standard scale, in which "excellent" meant complete relief from pain and no sign of bleeding after a single dose of the medicine and "good" meant pain relief and signs of improvement with a single dose, although additional doses may be needed for a complete resolution. In the first study, involving 73 patients aged 12 to 59 years, 249 bleeding episodes were treated with Rixubis. The effect of treatment in stopping bleeding episodes was judged to be excellent in 41% of cases and good in a further 55%. With reference to the prevention of bleeding, the average bleeding rate during treatment was 4.26 episodes a year, compared to an average of about 17 a year before enrollment in the study. A second study involved 23 children aged between just under 2 and almost 12, who experienced 26 bleeding episodes during the study: treatment of bleeding episodes was judged excellent in 50% of cases and good in one an additional 46%, while the average bleeding rate decreased from 6.8 to 2.7 episodes a year. In a third study, Rixubis was given to 14 patients who underwent surgery; treatment with Rixubis maintained the blood loss during surgery at the expected levels in patients without haemophilia B. The evaluation of these studies also indicated that the distribution of Rixubis in the body was similar to that of another product at factor IX base approved.
What is the risk associated with Rixubis - nonacog range?
The most common side effects with Rixubis (which may affect up to 1 in 10 people) are dysgeusia (taste disturbance) and pain in the limbs. Hypersensitivity (allergic) reactions may occur rarely and include angioedema (swelling of the tissues under the skin), burning and irritation at the injection site, chills, flushing, rash with itching, headache, hives, hypotension (low blood pressure), feeling tired or restless, nausea (feeling sick) or vomiting, tachycardia (rapid heartbeat), tightness of the chest, wheezing and tingling sensations. In some cases, the reactions become severe (anaphylaxis) and may be associated with a dangerous rapid drop in blood pressure. For the full list of all side effects reported with Rixubis, see the package leaflet. Rixubis should not be used in patients who are hypersensitive (allergic) to nonacog gamma or to any of the other ingredients of the medicine or with a known allergy to hamster proteins.
Why has Rixubis been approved - nonacog range?
The Agency's Committee for Medicinal Products for Human Use (CHMP) decided that Rixubis's benefits are greater than its risks and recommended that it be approved for use in the EU. The committee considered that Rixubis had been shown to be effective in preventing and treating bleeding episodes in adults and children with haemophilia B and was also effective in allowing them to undergo safe surgery. The safety profile was considered acceptable and was overcome by the beneficial effects.
What measures are being taken to ensure the safe and effective use of Rixubis - nonacog gamma?
A risk management plan has been developed to ensure that Rixubis 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 Rixubis, including the appropriate precautions to be followed by healthcare professionals and patients. Further information is available in the summary of the risk management plan.
More information on Rixubis - nonacog range
On 19 December 2014, the European Commission issued a marketing authorization for Rixubis, valid throughout the European Union. For more information about treatment with Rixubis, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist. Last update of this summary: 01-2015.
