Laronidase
What is Laronidase?[edit | edit source]
- Laronidase (Aldurazyme) is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme used for the treatment of Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I).
- Laronidase is a polymorphic variant of the human enzyme α‑L‑iduronidase that is produced by recombinant DNA technology in a Chinese hamster ovary cell line.
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]
- Pretreatment is recommended 60 minutes prior to the start of the infusion and may include antihistamines, antipyretics, or both.
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:
- infusion reactions such as pyrexia, chills, blood pressure increased, tachycardia, and oxygen saturation decreased
In 6 years and older are:
- rash, upper respiratory tract infection, injection site reaction, hyperreflexia, paresthesia, flushing, and poor venous access
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.
- Overdose related information is also available online at poisonhelp.org/help.
- In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.
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:
- BioMarin Pharmaceutical Inc.
- Novato, CA, USA
Distributed by:
- Genzyme Corporation
- Cambridge, MA, USA
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.
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