Aldurazyme (laronidase) is an enzyme replacement therapy indicated for pediatric and adult patients with Hurler, Hurler-Scheie, and moderate-to-severe Scheie forms of Mucopolysaccharidosis I (MPS I), demonstrating efficacy in improving respiratory function, mobility, and reducing glycosaminoglycan (GAG) accumulation.

Therapeutic Profile of Aldurazyme in Mucopolysaccharidosis I

Aldurazyme, a recombinant α-L-iduronidase, addresses the lysosomal enzyme deficiency in MPS I by hydrolyzing accumulated dermatan sulfate and heparan sulfate. Clinical trials (Studies 1–3) demonstrated statistically significant improvements in percent predicted forced vital capacity (FVC) and 6-minute walk distance (6MWT) compared to placebo, alongside sustained reductions in urinary GAG levels. These outcomes correlate with enhanced pulmonary function and physical endurance. However, CNS manifestations remain unaddressed, as the enzyme does not cross the blood-brain barrier. Efficacy in mildly symptomatic Scheie patients and long-term CNS impact are not established. Adverse effects predominantly involve infusion-related reactions (e.g., anaphylaxis, pyrexia), necessitating premedication and monitoring.