Idursulfase

Idursulfase Brand Name– Elaprase

What is Idursulfase

Idursulfase a purified form of human iduronate-2-sulfatase, is for the treatment of Hunter syndrome, which is also called mucopolysaccharidosis (MPS) II.

Patients with the syndrome do not produce enough of a functional lysosomal enzyme called iduronate-2-sulfatase because of a a genetic defect.

The deficiency of iduronate-2-sulfatase production causes an accumulation of glycosaminoglycans in cells throughout the body. The buildup of these carbohydrates produces a variety of physical and neurological problems including abnormal bone and joint growth, respiratory problems, learning disabilities, and hearing loss.

Many people with severe MPS II die during early adolescence, but people with milder forms of the disorder can live well into adulthood. Idursulfase serves as a replacement for the deficient enzyme in people with MPS II.

Idursulfase is produced by recombinant DNA technology in a human cell line and is the first treatment for Hunter syndrome.

A Hunter outcome survey has been established in order to understand better the variability and progression of Hunter syndrome in the population as a whole and to monitor and evaluate long-term treatment effects of idursulfase.

Patients and their physicians are encouraged to participate in this program. For more information, visit the official Elaprase website or call 1—866—888—0660.

Idursulfase was FDA-approved in July 2006.

Indications

  • mucopolysaccharidosis II (Hunter Syndrome)

For the treatment of mucopolysaccharidosis II (Hunter syndrome)

NOTE: Idursulfase has been designated as an orphan drug for this indication by the FDA.

Side Effects

  1. angioedema
  2. antibody formation
  3. arthralgia
  4. bronchospasm
  5. chills
  6. cough
  7. cyanosis
  8. diarrhea
  9. dizziness
  10. edema
  11. erythema
  12. fatigue
  13. fever
  14. flushing
  15. headache
  16. hypotension
  17. hypoxia
  18. infection
  19. infusion-related reactions
  20. nausea
  21. pruritus
  22. pulmonary embolism
  23. rash
  24. respiratory arrest
  25. seizures
  26. serious hypersensitivity reactions or anaphylaxis
  27. urticaria
  28. ventricular tachycardia
  29. vomiting
  30. wheezing

Monitoring Parameters

  • laboratory monitoring not necessary

Contraindications

  • acute bronchospasm
  • angioedema
  • asthma
  • breast-feeding
  • cardiac disease
  • children
  • fever
  • geriatric
  • heart failure
  • hypotension
  • infants
  • infusion-related reactions
  • neonates
  • pregnancy
  • pulmonary edema
  • respiratory depression
  • respiratory infection
  • respiratory insufficiency
  • serious hypersensitivity reactions or anaphylaxis

Interactions

There are no drug interactions associated with Idursulfase products.

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