Laronidase

Laronidase Brand Name– Aldurazyme

What is Laronidase

Laronidase (BM-101, recombinant human alpha-L-iduronidase) is an enzyme replacement therapy for the treatment of mucopolysaccharidosis I (MPS I), a rare, autosomal recessive lysosomal storage disorder caused by a deficiency of the enzyme alpha-L-iduronidase.

The lack of the enzyme causes glycosaminoglycans (GAG) to build up in cells; the manifestations can include growth and developmental delay, enlargement of spleen and liver, skeletal deformity, cardiac and pulmonary impairment, vision or hearing loss, and mental dysfunction.

The drug is indicated for enzyme replacement in patients with the Hurler and Hurler-Scheie forms of MPS I, and for Scheie patients with moderate to severe symptoms.

In a randomized, double-blinded, placebo-controlled study (n = 45), at 26 weeks, laronidase treatment statistically improved pulmonary function (FVC) by a mean of 5.6 percentage points (median 3; p =0.009); the 6-minute walk test distance, a measure of endurance, also improved by a mean of 38.1 meters (p =0.066).

An open-label study reported similar results.

The drug has also been shown to biochemically reduce the excess carbohydrates that are stored in the organs of patients with MPS I; however, the clinical significance of this carbohydrate reduction at the biochemical level is not certain.

Laronidase has not been evaluated to see if it improves the CNS manifestations (i.e., hydrocephalus, developmental delay) of the disorder.

Laronidase is the first drug treatment for MPS I; bone marrow transplantation being the only other viable option. Via a drug registry, the manufacturer will obtain long-term information related to the natural history of MPS I and the safety and efficacy of laronidase.

Patients are encouraged to learn more about the registry by visiting www.registrynxt.com or by calling 1-800-745-4447 (ext. 15500) in the US.

The manufacturer also offers CareConnectPSS (https://www.careconnectpss.com/), a program and service network designed to support people living with MPS I and their caregivers.

Indications

  • mucopolysaccharidosis I (MPS I)

For the treatment of the Hurler and Hurler-Scheie forms of mucopolysaccharidosis I (MPS I) and for treatment of the Scheie form of MPS I (moderate to severe symptoms only)

Side Effects

  1. abdominal pain
  2. angioedema
  3. antibody formation
  4. arthralgia
  5. back pain
  6. bradycardia
  7. bronchospasm
  8. chest pain (unspecified)
  9. chills
  10. corneal opacification
  11. cough
  12. diarrhea
  13. dyspnea
  14. edema
  15. fatigue
  16. fever
  17. flushing
  18. headache
  19. hyperbilirubinemia
  20. hyperhidrosis
  21. hyperreflexia
  22. hypotension
  23. hypoxia
  24. infection
  25. infusion-related reactions
  26. injection site reaction
  27. laryngeal edema
  28. musculoskeletal pain
  29. nausea
  30. pallor
  31. paresthesias
  32. peripheral edema
  33. pruritus
  34. rash
  35. serious hypersensitivity reactions or anaphylaxis
  36. sinus tachycardia
  37. tachypnea
  38. thrombocytopenia
  39. tremor
  40. urticaria
  41. vomiting
  42. wheezing

Monitoring Parameters

  • laboratory monitoring not necessary

Contraindications

  • breast-feeding
  • geriatric
  • infants
  • infusion-related reactions
  • laronidase hypersensitivity
  • neonates
  • pregnancy
  • pulmonary disease
  • serious hypersensitivity reactions or anaphylaxis

Interactions

There are no drug interactions associated with Laronidase products.

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