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Active Substance: idursulfase
Common Name: idursulfase
ATC Code: A16AB09
Marketing Authorisation Holder: Shire Human Genetic Therapies AB
Active Substance: idursulfase
Status: Authorised
Authorisation Date: 2007-01-08
Therapeutic Area: Mucopolysaccharidosis II
Pharmacotherapeutic Group: Other alimentary tract and metabolism products

Therapeutic Indication

Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (mucopolysaccharidosis II, MPS II). Heterozygous females were not studied in the clinical trials.

What is Elaprase?

Elaprase is a concentrate that is made up into a solution for infusion (drip) into a vein. It contains the active substance idursulfase.

What is Elaprase used for?

Elaprase is used to treat patients with Hunter syndrome. It is designed for long-term use.

Hunter syndrome, which is also known as mucopolysaccharidosis II, is a rare, inherited disease that primarily affects male patients. Patients with Hunter syndrome do not produce an enzyme called iduronate-2-sulfatase. This enzyme is needed to break down substances in the body called glycosaminoglycans (GAGs). Since patients with Hunter syndrome cannot break these substances down, the GAGs gradually build up in most of the organs in the body and damage them. This causes a wide range of symptoms, particularly difficulty breathing and difficulty walking. Without treatment, these symptoms become more severe over time.

Because the number of patients with Hunter syndrome is low, the disease is considered ‘rare’, and Elaprase was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 11 December 2001.

The medicine can only be obtained with a prescription.

How is Elaprase used?

Elaprase treatment should be supervised by a doctor or other healthcare professional who has experience in the management of patients with Hunter syndrome or other inherited diseases affecting the metabolism.

Elaprase is given every week, as an infusion into a vein, at a dose of 0.5 mg per kilogram body weight. The infusion should last three hours. However, as long as the patient does not develop an infusion reaction (rash, itching, fever, headache, high blood pressure or flushing), the medicine can be infused more rapidly, by gradually reducing the duration of infusion to one hour.

Patients who tolerate the infusions well for several months in a clinic may be able to start having them at home. Home infusions must be done under the supervision of a doctor or nurse.

How does Elaprase work?

The active substance in Elaprase, idursulfase, is a copy of the human enzyme iduronate-2-sulfatase It replaces the enzyme that is missing or defective in patients with Hunter syndrome. Supplying the enzyme will help to break down GAGs and stop it building up in the body thereby helping to improve the symptoms of the disease.

How has Elaprase been studied?

The main study of Elaprase involved 96 male patients aged between five and 31 years, and compared it with placebo (a dummy treatment). The main measures of effectiveness were lung function (‘forced vital capacity’, the maximum amount of air the patient could breathe out), and the distance the patients could walk in six minutes, which measures the combined effects of the illness on the heart, lungs, joints and other organs. These measurements were taken before and after a year of treatment.

What benefit has Elaprase shown during the studies?

Elaprase improved lung function and the walking ability of the patients. At the start of the study, the patients could walk an average of around 395 metres in six minutes. After a year, the patients receiving Elaprase could walk a further 43 metres on average, and the patients receiving placebo could walk a further 8 metres. The medicine also produced an improvement in lung function, while the patients on placebo showed a slight worsening.

What is the risk associated with Elaprase?

The most common side effects with Elaprase (seen in more than 1 patient in 10) are related to the infusion, including skin reactions (rash or itching), pyrexia (fever), headache, hypertension (high blood pressure) and swelling at the site of the infusion. Other side effects seen in more than 1 patient in 10 are flushing (reddening of the skin), wheezing, dyspnoea (difficulty breathing), abdominal pain (stomach ache), nausea (feeling sick), dyspepsia (heartburn), diarrhoea, vomiting and chest pain. Severe allergic reactions have been seen in some patients taking Elaprase. For the full list of all side effects reported with Elaprase, see the package leaflet.

Elaprase must not be used in people who have had a severe or life-threatening allergic (anaphylactic) reactions to idursulfase or any of the other ingredients if their allergy is not controllable. If given to patients who have had severe allergic reactions in the past, Elaprase should be used with caution, and trained staff and equipment for emergency resuscitation should be available during infusion.

Why has Elaprase been approved?

The CHMP concluded that the improvements shown in the study, even if limited, represent a clinical benefit in the treatment of Hunter syndrome. The Committee decided that Elaprase’s benefits are greater than its risks and recommended that it be given marketing authorisation.

Elaprase has been authorised under 'Exceptional Circumstances'. This means that because Hunter syndrome is rare, it has not been possible to obtain complete information about Elaprase. Every year, the European Medicines Agency will review any new information that may become available and this summary will be updated as necessary.

What information is still awaited for Elaprase?

The company that makes Elaprase will investigate the long-term effects of the medicine, and whether it stimulates the body to produce antibodies that could affect the medicine’s safety and effectiveness. The company will also study the medicine’s effectiveness in patients below five years of age, and investigate whether it has any effects on the lungs, the heart or the blood vessels.

What measures are being taken to ensure the safe and effective use of Elaprase?

A risk management plan has been developed to ensure that Elaprase 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 Elaprase, including the appropriate precautions to be followed by healthcare professionals and patients.

Other information about Elaprase

The European Commission granted a marketing authorisation valid throughout the European Union for Elaprase on 8 January 2007.

For more information about treatment with Elaprase, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

Source: European Medicines Agency

Disclaimer: Every effort has been made to ensure that the information provided here is accurate, up-to-date and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. This information has been compiled for use by healthcare practitioners and consumers in the United States. The absence of a warning for a given drug or combination thereof in no way should be construed to indicate that the drug or combination is safe, effective or appropriate for any given patient. If you have questions about the substances you are taking, check with your doctor, nurse or pharmacist.