What is Elaprase?
Elaprase is a concentrate for solution for infusion that contains the active substance idursulfase.
What is Elaprase used for?
Elaprase is used to treat patients with Hunter syndrome and is intended for long-term use.
Hunter syndrome, also known as mucopolysaccharidosis II, is a rare inherited disorder that primarily affects males. Patients suffering from this disorder do not produce an enzyme called iduronate-2-sulphatase, which serves to break down substances in the body called glycosaminoglycans (GAGs). As a result, GAGs gradually accumulate in most of these patients' organs, damaging them. This causes a wide range of symptoms, particularly difficulty breathing and difficulty walking. In the absence of treatment, these symptoms become increasingly severe with the passage of time.
Because the number of patients with Hunter syndrome is small, the disease is considered rare and on 11 December 2001 Elaprase was designated an "orphan medicine" (a medicine used to treat rare diseases).
The medicine can only be obtained with a prescription.
How is Elaprase used?
Elaprase is given every week as an intravenous infusion (drip into a vein). The dose for adults, children and adolescents is 0.5 mg / kg of body weight. The appropriate dose of Elaprase must be diluted in a saline solution before being infused.
The infusion should last 3 hours, which can be gradually reduced to 1 hour, provided that the patient does not manifest infusion reactions (rash, itching, fever, headache, hypertension or redness).
How does Elaprase work?
The active substance in Elaprase, idursulfase, is a copy of the human enzyme iduronate-2-sulphatase. is
produced by a method known as "recombinant DNA technology": the enzyme is produced by a human cell in which a gene (DNA) has been introduced which makes it capable of producing the enzyme. Idursulfase replaces the missing or insufficient enzyme in patients with Hunter syndrome. By providing the enzyme, you can improve or control the symptoms of the disease.
What studies have been carried out on Elaprase?
The effects of Elaprase were first tested in experimental models before being studied in humans.
The main study of Elaprase was carried out on 96 male patients aged between 5 and 31 years and the efficacy of the medicine was compared with that of a placebo (a dummy treatment). The main measures of effectiveness were pulmonary function ("forced vital capacity", the maximum amount of air the patient is able to emit when breathing) and the distance that the patients can walk along in 6 minutes, which measures the effects joint of the disease on the heart, lungs, joints and other organs. These measures were taken before treatment and after 52 weeks of treatment.
What benefit has Elaprase shown during the studies?
The study showed that Elaprase improves both lung function and patients' ability to walk. After 52 weeks, patients treated with Elaprase could travel 43.3 m in 6 minutes, while those treated with placebo only 8.2 m. The medicine also produced an improvement in lung function, while a slight deterioration was found in patients treated with placebo.
What is the risk associated with Elaprase?
The most common side effects of Elaprase (seen in more than 1 patient in 10) are infusion-related reactions, such as skin reactions (rash or itching), pyrexia (fever), headache, hypertension (high blood pressure) and swelling at the infusion site. Other very common side effects were dyspepsia (heartburn) and chest pain. For the full list of all side effects reported with Elaprase, see the Package Leaflet.
Elaprase must not be used in people who are hypersensitive (allergic) to idursulfase or to any of the excipients.
Why has Elaprase been approved?
The Committee for Medicinal Products for Human Use (CHMP) concluded that the improvements shown by the study, however modest, represent a clinical benefit in the treatment of
Hunter decided that Elaprase's benefits are greater than its risks in the long-term treatment of patients with Hunter syndrome and recommended that Elaprase be given marketing authorization.
Elaprase has been authorized "in exceptional circumstances" because, since Hunter syndrome is a rare disease, more detailed information on the medicine could not be obtained. The European Medicines Agency (EMEA) will review any new information available each year and this summary will be updated as necessary.
What information is still awaited for Elaprase?
The company that makes Elaprase will examine the long-term effects of the medicine and investigate to see if it stimulates the body to produce antibodies (special proteins) against the medicine. The company will also examine the efficacy of the medicine in patients under the age of 5 and will investigate to find out if there are any effects on the lungs, heart or blood vessels.
What measures are being taken to ensure the safe use of Elaprase?
The company that makes Elaprase will check the safety of the medicine with a long-term investigation of patients with Hunter syndrome, which will include an analysis of the effects of the drug in female patients and a study to determine whether it can be used in a way safe at the patients' home.
More information on Elaprase:
On 8 January 2007, the European Commission issued a marketing authorization for Elaprase, valid throughout the European Union, to Shire Human Genetic Therapies AB.
To register Elaprase as an orphan medicinal product, click here.
For the full EPAR of Elaprase, click here.
Last update of this summary: 11-2006.
