Abstract
This study assessed the efficacy and safety of a biosimilar Laronidase (CinnaGen Company, Iran), compared to the reference Laronidase (Aldurazyme, BioMarin, USA) in maintaining urinary glycosaminoglycan (uGAG) levels in mucopolysaccharidosis type I (MPS I) patients. In this phase III, open-label, single-sequence, and cross-over study, MPS I patients received Aldurazyme for 12 weeks, followed by Laronidase (CinnaGen) for another 12 weeks. The primary outcome was the assessment of mean uGAG levels at the final visits of each medication administration. The secondary outcomes included the assessment of 6-minute walking test (6MWT), predicted forced vital capacity (FVC), enzyme activity assay, and adverse events (AEs). Twelve patients with mean (standard deviation [SD]) age of 10.25 ± 3.11 years were included in the study. The mean (± SD) uGAG level was 220.47 ± 177.13 CPC unit/g creatinine at the end of Aldurazyme treatment and changed to 270.02 ± 111.55 CPC unit/g creatinine at the end of Laronidase (CinnaGen) treatment, with variable patient responses. The mean 6MWT and predicted FVC improved by almost 30 m and 13%, respectively. Post-infusion enzyme activity levels showed similar patterns between Aldurazyme and Laronidase (CinnaGen), with no detectable activity before the infusion. A total of 30 AEs were reported during the trial. In the first time period (receiving Aldurazyme), AEs were reported for seven patients (58.33%), and in the subsequent time period (receiving Laronidase (CinnaGen)), AEs were reported for six patients (50%). Among all, nasopharyngitis was the most common reported AE. In conclusion, the biosimilar Laronidase was shown to have a comparable efficacy and safety profile compared with the reference Laronidase.Clinical Trial Registration: Clinicaltrials.gov: NCT06406153; First registration: 09/05/2024.