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
- abdominal pain
- angioedema
- antibody formation
- arthralgia
- back pain
- bradycardia
- bronchospasm
- chest pain (unspecified)
- chills
- corneal opacification
- cough
- diarrhea
- dyspnea
- edema
- fatigue
- fever
- flushing
- headache
- hyperbilirubinemia
- hyperhidrosis
- hyperreflexia
- hypotension
- hypoxia
- infection
- infusion-related reactions
- injection site reaction
- laryngeal edema
- musculoskeletal pain
- nausea
- pallor
- paresthesias
- peripheral edema
- pruritus
- rash
- serious hypersensitivity reactions or anaphylaxis
- sinus tachycardia
- tachypnea
- thrombocytopenia
- tremor
- urticaria
- vomiting
- 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.