Presentation and Status in Health Basket
This treatment should be supervised by a physician or other healthcare professional experienced in the management of patients with MPS II disease or other inherited metabolic disorders.
Elaprase is administered at a dose of 0.5 mg/kg body weight every week by intravenous infusion over a 3 hour period, which may be gradually reduced to 1 hour if no infusion-associated reactions are observed.
Infusion at home may be considered for patients who have received several months of treatment in the clinic and who are tolerating their infusions well. Home infusions should be performed under the surveillance of a physician or other healthcare professional.
Elderly patients: There is no clinical experience in patients over 65 years of age.
Patients with renal or hepatic impairment: There is no clinical experience in patients with renal or hepatic insufficiency.
Paediatric population: The dose for children and adolescents is the same as for adults, 0.5 mg/kg body weight weekly.
Method of administration: For instructions on dilution of the medicinal product before administration see section 6.6 at the attached doctor’s leaflet.
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.
Severe or life-threatening hypersensitivity to the active substance or to any of the excipients if hypersensitivity is not controllable.
Infusion-related reactions: Patients treated with idursulfase may develop infusion-related reactions. During clinical trials, the most common infusion-related reactions included cutaneous reactions (rash, pruritus, urticaria), pyrexia, headache, hypertension, and flushing. Infusion-related reactions were treated or ameliorated by slowing the infusion rate, interrupting the infusion, or by administration of medicinal products, such as antihistamines, antipyretics, low-dose corticosteroids (prednisone and methylprednisolone), or beta-agonist nebulisation. No patient discontinued treatment due to an infusion reaction during clinical studies.
Special care should be taken when administering an infusion in patients with severe underlying airway disease. These patients should be closely monitored and infused in an appropriate clinical setting. Caution must be exercised in the management and treatment of such patients by limitation or careful monitoring of antihistamine and other sedative medicinal product use. Institution of positive-airway pressure may be necessary in some cases.
Delaying the infusion in patients who present with an acute febrile respiratory illness should be considered. Patients using supplemental oxygen should have this treatment readily available during infusion in the event of an infusion-related reaction.
Anaphylactoid/anaphylactic reactions: Anaphylactoid/anaphylactic reactions, which have the potential to be life threatening, have been observed in some patients treated with idursulfase up to several years after initiating treatment. Late emergent symptoms and signs of anaphylactoid/anaphylactic reactions have been observed as long as 24 hours after an initial reaction. If an anaphylactoid/anaphylactic reaction occurs the infusion should be immediately suspended and appropriate treatment and observation initiated. The current medical standards for emergency treatment are to be observed. Patients experiencing severe or refractory anaphylactoid/anaphylactic reactions may require prolonged clinical monitoring. Patients who have experienced anaphylactoid/anaphylactic reactions should be treated with caution when re-administering idursulfase, appropriately trained personnel and equipment for emergency resuscitation (including epinephrine) should be available during infusions. Severe or potentially life-threatening hypersensitivity is a contraindication to rechallenge, if hypersensitivity is not controllable.
Patients with the complete deletion/large rearrangement genotype: Paediatric patients with the complete deletion/large rearrangement genotype have a high probability of developing antibodies, including neutralizing antibodies, in response to exposure to idursulfase. Patients with this genotype have a higher probability of developing infusion-related adverse events and tend to show a muted response as assessed by decrease in urinary output of glycosaminoglycans, liver size and spleen volume compared to patients with the missense genotype. Management of patients must be decided on an individual basis.
Sodium: This medicinal product contains 0.482 mmol sodium (or 11.1 mg) per vial. This is equivalent to 0.6% of the WHO recommended maximum daily intake of 2 g sodium for an adult.
Traceability: In order to improve the traceability of biological medicinal products, the name and batch number of the administered product should be clearly recorded.
The most common adverse reaction were infusion-related reactions included cutaneous reactions (rash, pruritus, urticaria, and erythema), pyrexia, flushing, wheezing, dyspnoea, headache, vomiting, abdominal pain, nausea and chest pain. The frequency of infusion-related reactions decreased over time with continued treatment.
See prescribing information for full details.
No formal medicinal product interaction studies have been conducted with idursulfase.
Based on its metabolism in cellular lysosomes, idursulfase would not be a candidate for cytochrome P450 mediated interactions.
Pregnancy and Lactation
Pregnancy: There are no data or limited amount of data from the use of idursulfase in pregnant women. Animal studies do not indicate direct or indirect harmful effects with respect to reproductive toxicity. As a precautionary measure, it is preferable to avoid the use of idursulfase during pregnancy.
Lactation: It is not known whether idursulfase is excreted in human breast milk. Available data in animals have shown excretion of idursulfase in milk. A risk to the newborns/infants cannot be excluded. A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from idursulfase therapy taking into account the benefit of breast feeding for the child and the benefit of therapy for the woman.
There is no experience with overdoses of Elaprase.
Storage: Store in a refrigerator (2°C – 8°C). Do not freeze.