Laronidase

From WikiMD's Wellness Encyclopedia

What is Laronidase?[edit | edit source]



What are the uses of this medicine?[edit | edit source]

  • Laronidase (Aldurazyme) is used for adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms.

Limitations of Use:

  • The risks and benefits of treating mildly affected patients with the Scheie form have not been established.
  • Aldurazyme cannot be used for effects on the central nervous system manifestations of the disorder.


How does this medicine work?[edit | edit source]


Mucopolysaccharide storage disorders:

  • Mucopolysaccharide storage disorders are caused by the deficiency of specific lysosomal enzymes required for the catabolism of glycosaminoglycans (GAG).
  • Mucopolysaccharidosis I (MPS I) is characterized by the deficiency of α-L-iduronidase, a lysosomal hydrolase which catalyzes the hydrolysis of terminal α-L-iduronic acid residues of dermatan sulfate and heparan sulfate.
  • Reduced or absent α-L-iduronidase activity results in the accumulation of the GAG substrates, dermatan sulfate and heparan sulfate, throughout the body and leads to widespread cellular, tissue, and organ dysfunction.
  • Aldurazyme therapy in MPS I is to provide exogenous enzyme for uptake into lysosomes and increase the catabolism of GAG.
  • Aldurazyme uptake by cells into lysosomes is most likely mediated by the mannose-6-phosphate-terminated oligosaccharide chains of laronidase binding to specific mannose-6-phosphate receptors.


Who Should Not Use this medicine ?[edit | edit source]

Limitations of Use:

  • The risks and benefits of treating mildly affected patients with the Scheie form have not been established.
  • Aldurazyme cannot be used for effects on the central nervous system manifestations of the disorder.


What drug interactions can this medicine cause?[edit | edit source]

  • Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.


Is this medicine FDA approved?[edit | edit source]

  • Initial U.S. Approval: 2003


How should this medicine be used?[edit | edit source]

Recommended dosage:

  • The recommended dosage is 0.58 mg/kg of body weight administered once weekly as an intravenous infusion.

Administration:

  • Aldurazyme must be administered once weekly as an intravenous infusion.
  • Aldurazyme does not contain any preservatives; therefore, after dilution with saline, the infusion bags should be used immediately.
  • If immediate use is not possible, the diluted solution should be stored refrigerated at 2°C to 8°C (36°F to 46°F) for up to 36 hours.
  • Aldurazyme must not be administered with other medicinal products in the same infusion.


What are the dosage forms and brand names of this medicine?[edit | edit source]

This medicine is available in fallowing doasage form:

  • As Injection: 2.9 mg/5 mL (0.58 mg/mL) of laronidase in a single-dose vial

This medicine is available in fallowing brand namesː

  • Aldurazyme


What side effects can this medication cause?[edit | edit source]

The most common side effects of this medicine include: In 6 months of age and older are:

In 6 years and older are:


What special precautions should I follow?[edit | edit source]

  • Life-threatening anaphylactic reactions have been observed in some patients during Aldurazyme infusion and up to 3 hours after infusion. Appropriate medical support and monitoring measures should be readily available when Aldurazyme is administered. If anaphylactic or other severe hypersensitivity reactions occur, immediately discontinue the infusion and initiate appropriate treatment, which may include ventilatory support, treatment with inhaled beta-adrenergic agonists, epinephrine, and IV corticosteroids.
  • Patients with acute febrile or respiratory illness at the time of Aldurazyme infusion may be at greater risk for infusion reactions. Consider delaying Aldurazyme infusion. Sleep apnea is common in MPS I patients. Evaluation of airway patency should be considered prior to initiation of treatment with Aldurazyme. Appropriate respiratory support should be available during infusion.
  • Caution should be exercised when administering Aldurazyme to patients susceptible to fluid overload. Consider a decreased total infusion volume and infusion rate when administering Aldurazyme to these patients. Appropriate medical monitoring and support measures should be available during infusion.
  • Pretreatment is recommended prior to the infusion to reduce the risk of infusion reactions and may include antihistamines, antipyretics, or both. If infusion reactions occur, decreasing the infusion rate, temporarily stopping the infusion, or administering additional antipyretics and/or antihistamines may ameliorate the symptoms.
  • There are no available data on the presence of laronidase in human milk or the effects on milk production.


What to do in case of emergency/overdose?[edit | edit source]

Symptoms of overdose may include:

  • There have been no reports of overdose with Aldurazyme.

Management of overdosage:

  • In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.


Can this medicine be used in pregnancy?[edit | edit source]

  • Available data from published case reports and postmarketing experience with Aldurazyme use in pregnant women are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes.


Can this medicine be used in children?[edit | edit source]

  • The safety and effectiveness of Aldurazyme was assessed in pediatric patients aged 6 months to 5 years old, and was found to be similar to the safety and effectiveness of Aldurazyme in pediatric patients 6 to 18 years, and adults.


What are the active and inactive ingredients in this medicine?[edit | edit source]

Active ingredients:

  • LARONIDASE

Inactive Ingredients:

  • none


Who manufactures and distributes this medicine?[edit | edit source]

Manufactured by:

Distributed by:


What should I know about storage and disposal of this medication?[edit | edit source]

  • Store refrigerated at 2-8°C (36-46°F).
  • Do not freeze or shake.


Laronidase Resources
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