Abstract | BACKGROUND AND OBJECTIVE: METHODS: A phase III/IV, open-label, single-arm, multicenter safety study was conducted in Canadian patients with Fabry disease between August 2011 and September 2017 as a regulatory requirement to assess the safety of agalsidase alfa produced using an animal component-free bioreactor process. Eligible patients had a documented diagnosis of Fabry disease and satisfied current Canadian guidelines for receiving enzyme replacement therapy for Fabry disease. Following treatment with animal component-free bioreactor-processed agalsidase alfa, treatment-emergent adverse events were monitored, and post hoc analyses of infusion-related reactions by antidrug antibody and neutralizing antibody statuses were conducted. The data were analyzed using descriptive statistics. RESULTS: A total of 167 patients (mean [standard deviation] age, 48.9 [14.8] years), including six pediatric patients (< 18 years of age), received at least one full or partial infusion of agalsidase alfa animal component-free. Fewer than 5% of treatment-emergent adverse events (212/4446) observed in 40 patients were reported as infusion-related reactions. Antidrug antibody and neutralizing antibody status did not affect the proportion of patients with infusion-related reactions. No clinically significant changes in vital signs were observed in patients over the course of the study. CONCLUSIONS: Long-term treatment with bioreactor-produced agalsidase alfa animal component-free did not reveal new safety signals in this population of Canadian patients with Fabry disease. The treatment-emergent adverse event profile was consistent with the clinical manifestations of the disease and the known safety profile of roller bottle-produced agalsidase alfa. CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov identifier NCT01298141.
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Authors | Aneal Khan, Sandra M Sirrs, Daniel G Bichet, Chantal F Morel, Adina Tocoian, Lan Lan, Michael L West, Canadian Fabry Disease Initiative |
Journal | Drugs in R&D
(Drugs R D)
Vol. 21
Issue 4
Pg. 385-397
(Dec 2021)
ISSN: 1179-6901 [Electronic] New Zealand |
PMID | 34542871
(Publication Type: Journal Article, Multicenter Study)
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Copyright | © 2021. The Author(s). |
Chemical References |
- Isoenzymes
- Recombinant Proteins
- agalsidase alfa
- alpha-Galactosidase
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Topics |
- Animals
- Bioreactors
- Canada
- Child
- Enzyme Replacement Therapy
- Fabry Disease
(drug therapy)
- Humans
- Isoenzymes
- Middle Aged
- Quality of Life
- Recombinant Proteins
(therapeutic use)
- Treatment Outcome
- alpha-Galactosidase
(adverse effects)
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