210589-09-6

Basic information

• Product Name: Laronidase [usan:inn]
• Synonyms: Aldurazyme;alpha-L-Iduronidase;Ec 3.2.1.76;Human recombinant alpha-L-iduronidase;Iduronidase, alpha-L-(8-histidine) (human);Laronidase;Laronidase [usan:inn];ANTI-IDUA (CENTER) antibody produced in rabbit
• CAS NO: 210589-09-6
• Mol File: 210589-09-6.mol
• Article Data: 0

Chemical Properties

• Appearance: /
• Storage Temp.: -20°C
• CAS DataBase Reference: Laronidase [usan:inn](CAS DataBase Reference)
• NIST Chemistry Reference: Laronidase [usan:inn](210589-09-6)
• EPA Substance Registry System: Laronidase [usan:inn](210589-09-6)

Safety Data

• Hazardous Substances Data: 210589-09-6(Hazardous Substances Data)

Usage

Description

Laronidase [usan:inn], also known as Aldurazyme, is a recombinant form of the human alpha-L-iduronidase enzyme produced by overexpression in a Chinese hamster ovary cell line. It is used as an enzyme replacement therapy for the treatment of patients with Mucopolysaccharidosis I (MPS I), a rare genetic lysosomal storage disease caused by the deficiency of alpha-L-iduronidase.

Uses

Used in Enzyme Replacement Therapy for Mucopolysaccharidosis I (MPS I):
Laronidase [usan:inn] is used as an enzyme replacement therapy for the treatment of patients with Hurler and Hurler-Scheie syndromes and patients with the Scheie syndrome who have moderate to severe symptoms. It helps in the degradation of dermatan sulfate and heparin sulfate, which accumulate in various tissues due to the deficiency of alpha-L-iduronidase enzyme in MPS I patients. The recommended dosage regimen of laronidase is 0.58 mg/kg of body weight administered once weekly as an intravenous infusion.
Clinical trials have demonstrated the efficacy of laronidase in improving pulmonary function and endurance, with laronidase-treated patients showing a mean increase of 4.0% in predicted forced vital capacity (FVC) and a mean increase of 38.0 m in the distance walked in 6 minutes as compared with placebo-treated patients. Additionally, reductions in liver size and urinary glycosaminoglycan excretion were observed.
The most common adverse events associated with laronidase are upper respiratory tract infection, rash, and injection site reaction.

Originator

BioMarin (US)