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Clinical Trial
. 2025 Aug 19;15(1):30427.
doi: 10.1038/s41598-025-16351-4.

Efficacy and safety of a biosimilar laronidase versus the reference laronidase in patients with mucopolysaccharidosis type I

Ali Rabbani  1 , Mohammadreza Alaei  2 , Samaneh Norouzi Asl  3   4 , Aria Setoodeh  5 , Marjan Shakiba  6 , Shadab Salehpour  7 , Peyman Eshraghi  4   8 , Omid Salehpour  9 , Araz Sabzvari  10 , Hamidreza Kafi  11 , Ali Jarollahi  12
Affiliations
Clinical Trial

Efficacy and safety of a biosimilar laronidase versus the reference laronidase in patients with mucopolysaccharidosis type I

Ali Rabbani et al. Sci Rep. .

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.

Keywords: Aldurazyme; Laronidase; Mucopolysaccharidosis; Urinary glycosaminoglycans.

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Conflict of interest statement

Competing interests: Hamidreza Kafi is the head of the medical department of Orchid Pharmed Company which is in collaboration with CinnaGen company with respect to conducting clinical trials. Araz Sabzvari is a member of CinnaGen medical biotechnology research center. This research center collaborates with universities and researchers all over the world with regards to research and development of medications and health issues. Other authors have no conflict of interest to declare. Ethics approval: This study was performed in line with the principles of the Declaration of Helsinki and its later amendments, and with Good Clinical Practice guidelines. Approval was granted by the ethics committee of Tehran University of Medical Sciences (IR.TUMS.TIPS.REC.1401.001). Informed consent: Written informed consent was obtained from all legal guardians of the patients.

Figures

Fig. 1
Fig. 1
CONSORT flow diagram of the study.
Fig. 2
Fig. 2
The urinary glycosaminoglycan (uGAG) levels per patient.
See this image and copyright information in PMC

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