What is Rixubis and
what is it used for?
Rixubis is a medicine
used to treat and prevent bleeding in patients with haemophilia B, an inherited
bleeding disorder caused by lack of factor IX. It can be used in patients of
all ages, and for short-term or long-term use. Rixubis contains the active substance nonacog gamma.
How is Rixubis used?
Rixubis can only be
obtained with a prescription and treatment should be started under the
supervision of a doctor who has experience in the treatment of haemophilia.
Rixubis is available
as a powder and solvent that are mixed together to make a solution for
injection into a vein. The dose and frequency of treatment depend on the
patient’s bodyweight and whether Rixubis is used to treat or prevent bleeding,
as well as the seriousness of the haemophilia, the extent and location of the
bleeding and the patient’s age and health. For further information, see
the summary of product characteristics (also part of the
EPAR).
Patients or their
carers may be able to administer Rixubis themselves at home once they have been
trained appropriately. For full details, see the package leaflet.
How does Rixubis
work?
Patients with
haemophilia B lack factor IX, which is needed for blood to clot properly. This
lack causes blood 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 clot in the same way. Rixubis can
therefore be used to replace the missing factor IX, giving temporary control of
the bleeding disorder.
Nonacog gamma is not
extracted from human blood but is made by a method known as ‘recombinant DNA
technology’: it is made by hamster cells into which a gene (DNA) has been
introduced that makes the cells able to produce the human clotting factor.
What benefits of
Rixubis have been shown in studies?
The benefits of
Rixubis in treating and preventing bleeding episodes have been shown in three
main studies involving patients with severe or moderately severe haemophilia B.
None of the studies compared the effectiveness of Rixubis directly with another
medicine. Effectiveness in stopping bleeding was measured on a standard scale
in which ‘excellent’ meant complete relief of pain and no signs of bleeding
after a single dose of the medicine, and ‘good’ meant relief of pain and signs
of improvement with a single dose, although further doses might be needed for
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 rated
excellent in 41% of cases and good in a further 55%. With respect to prevention
of bleeding, the average bleeding rate during treatment was 4.26 bleeds a year,
compared with an average of about 17 per year before enrolment. A second study
involved 23 children aged from just under 2 to nearly 12 years of age, who
experienced 26 bleeding episodes during the study: treatment of bleeding
episodes was rated as excellent in 50% of cases and good in another 46%, and
the average bleeding rate was reduced from 6.8 to 2.7 bleeds per year. In a
third study, Rixubis was given to 14 patients undergoing surgery; treatment
with Rixubis kept blood loss during the surgery to levels expected in patients
without haemophilia B.
The evaluation of
these studies also indicated that Rixubis was distributed in the body similarly
to another approved factor IX product.
What are the risks
associated with Rixubis?
The most common side
effects with Rixubis (which may affect up to 1 in 10 people) are dysgeusia
(taste disturbances) and pain in the limbs. Hypersensitivity (allergic)
reactions may occur rarely, and can include angioedema (swelling of tissues
under the skin), burning and stinging at the injection site, chills, flushing,
itchy rash, 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,
reactions become severe (anaphylaxis) and may be associated with dangerously
steep falls in blood pressure. For the full list of all side effects with
Rixubis see the package leaflet.
Rixubis must not be
used in patients who are hypersensitive (allergic) to nonacog gamma or any of
its other ingredients, or who are known to be allergic to hamster protein.
Why is Rixubis
approved?
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 surgery safely. The safety profile
was considered acceptable and was outweighed by the beneficial effects.
What measures are
being taken to ensure the safe and effective use of Rixubis?
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
can be found in the
Other information
about Rixubis
