Mastodon

Elaprase® (Concentrate) Instructions for Use

ATC Code

A16AB09 (Idursulfase)

Active Substance

Idursulfase (Rec.INN registered by WHO)

Clinical-Pharmacological Group

Drug for the treatment of hereditary enzymatic deficiency

Pharmacotherapeutic Group

Drugs for the treatment of digestive tract and metabolism disorders – enzymes

Pharmacological Action

A means for enzyme replacement therapy. It is a human lysosomal enzyme iduronate-2-sulfatase, which is produced using recombinant DNA technology on a human cell line.

Idursulfase is an enzyme that hydrolyzes 2-sulfate esters, which are intermediate products of the breakdown of glycosaminoglycans – dermatan sulfate and heparan sulfate in the lysosomes of various cell types.

Idursulfase has been shown to improve walking ability in patients with mucopolysaccharidosis type 2 (Hunter syndrome).

Pharmacokinetics

With a single infusion over 1 hour, the AUC value of idursulfase is directly proportional to the dose in the dose range from 0.15 mg/kg to 1.5 mg/kg. Cmax – 1.5 µg/ml, T1/2 – 44 min, clearance – 3 ml/min/kg.

Indications

  • Mucopolysaccharidosis type 2 (Hunter syndrome) for long-term enzyme replacement therapy.

Use only after confirmation of diagnosis by biochemical or genetic testing.

ICD codes

ICD-10 code Indication
E76 Disorders of glycosaminoglycan metabolism
ICD-11 code Indication
5C56.3Z Mucopolysaccharidosis, unspecified

Dosage Regimen

The method of application and dosage regimen for a specific drug depend on its form of release and other factors. The optimal dosage regimen is determined by the doctor. It is necessary to strictly adhere to the compliance of the dosage form of a specific drug with the indications for use and dosage regimen.

Administer 0.5 mg per kilogram of body weight as an intravenous infusion once every week.

Dilute the concentrate before use. Initiate the infusion at a rate of 8 ml per hour for the first 15 minutes. If well tolerated, increase the rate to 100 ml per hour to complete the infusion over approximately 3 hours.

Do not administer as an intravenous bolus. Adjust the infusion rate based on individual patient tolerance.

Adverse Reactions

Observe for infusion-associated reactions, which are very common. These include headache, fever, urticaria, hypertension, arthralgia, and dyspnea.

Monitor for signs of severe hypersensitivity reactions, including anaphylaxis. Antibody formation against the enzyme is common; clinical significance is uncertain.

Other frequently reported reactions involve skin, musculoskeletal system, and general disorders. Manage reactions with premedication and infusion rate adjustment.

Contraindications

  • Do not use in patients with a known history of severe hypersensitivity reactions, including anaphylaxis, to idursulfase or any excipient in the formulation.

There are no other absolute contraindications when used for the approved indication.

Drug Interactions

Formal drug interaction studies have not been conducted.

Exercise caution when administering with other medicinal products known to cause infusion-related reactions or having the potential to induce hypersensitivity.

The risk of additive adverse effects should be considered. Monitor patients closely when co-administering such agents.

Overdose

No specific antidote is known. Doses up to 3 mg/kg have been administered in clinical studies without unexpected adverse effects.

In case of suspected overdose, discontinue the infusion immediately and institute supportive and symptomatic treatment.

Closely monitor the patient’s vital signs and clinical status. Manage any resulting infusion reactions aggressively.

Use in Pregnancy and Lactation

The safety of use during pregnancy has not been studied. It is not known whether Idursulfase is excreted in human breast milk.

Pediatric Use

The safety and efficacy of Idursulfase use in children under 5 years of age have not been studied.

Special Precautions

During the administration of Idursulfase, there is a high risk of developing life-threatening anaphylactic reactions, so the infusion should be carried out in conditions that allow for emergency therapy and clinical monitoring.

After relief of an anaphylactic reaction during or immediately after the infusion, a repeated development of an anaphylactic reaction is possible 24 hours after the infusion. Therefore, patients who experienced initial infusion reactions require longer medical supervision.

If infusion reactions develop in connection with the first infusion, premedication with antihistamines should be performed before or during subsequent infusions.

The safety and efficacy of idursulfase use in children under 5 years of age have not been studied.

Storage Conditions

Store at 2°C (36°F) to 25°C (77°F). Keep in original packaging, protected from light. Keep out of reach of children.

Dispensing Status

Rx Only

Important Safety Information

This information is for educational purposes only and does not replace professional medical advice. Always consult your doctor before use. Dosage and side effects may vary. Use only as prescribed.

Medical Disclaimer

Brand (or Active Substance), Marketing Authorisation Holder, Dosage Form

Marketing Authorization Holder

Shire Human Genetic Therapies, Inc. (USA)

Manufactured By

Baxter Healthcare Corp. (USA)

Or

Cangene BioPharma LLC (USA)

Dosage Form

Bottle Rx Icon Elaprase® Concentrate for solution for infusion 6 mg/3 ml: vial 1, 4, or 10 pcs.

Dosage Form, Packaging, and Composition

Concentrate for solution for infusion colorless, transparent or slightly opalescent.

1 ml 1 vial
Idursulfase 2 mg 6 mg

Excipients : sodium chloride, sodium hydrogen phosphate heptahydrate, sodium dihydrogen phosphate monohydrate, polysorbate 20, water for injections.

3 ml – vials of colorless glass (type I) with a capacity of 5 ml (1) – cardboard boxes.
3 ml – vials of colorless glass (type I) with a capacity of 5 ml (4) – cardboard boxes.
3 ml – vials of colorless glass (type I) with a capacity of 5 ml (10) – cardboard boxes.

Marketing Authorization Holder

Takeda Pharmaceuticals International AG Ireland Branch (Ireland)

Manufactured By

Vetter Pharma-Fertigung, GmbH & Co. KG (Germany)

Labeled By

DHL SUPPLY CHAIN (NETHERLANDS) B.V. (Netherlands)

Quality Control Release

SHIRE PHARMACEUTICALS IRELAND, Limited (Ireland)

Dosage Form

Bottle Rx Icon Elaprase® Concentrate for solution for infusion 2 mg/1 ml: fl. 1, 4 or 10 pcs.

Dosage Form, Packaging, and Composition

Concentrate for solution for infusion in the form of a colorless, transparent or slightly opalescent solution.

1 ml
Idursulfase 2 mg

Excipients : sodium chloride – 8 mg, sodium hydrogen phosphate heptahydrate – 0.99 mg, sodium dihydrogen phosphate monohydrate – 2.25 mg, polysorbate 20 – 0.0002 ml, water for injections – up to 1 ml.

3 ml – vials of colorless glass (type I) with a capacity of 5 ml (1) – cardboard boxes.
3 ml – vials of colorless glass (type I) with a capacity of 5 ml (4) – cardboard boxes.
3 ml – vials of colorless glass (type I) with a capacity of 5 ml (10) – cardboard boxes.

Table of Contents

→ TABLE OF CONTENTS