Journal article
Oral migalastat HCl leads to greater systemic exposure and tissue levels of active α-galactosidase A in fabry patients when co-administered with infused agalsidase
DG Warnock, DG Bichet, M Holida, O Goker-Alpan, K Nicholls, M Thomas, F Eyskens, S Shankar, M Adera, S Sitaraman, R Khanna, JJ Flanagan, BA Wustman, J Barth, C Barlow, KJ Valenzano, DJ Lockhart, P Boudes, FK Johnson
Plos One | Published : 2015
Abstract
Migalastat HCl (AT1001, 1-Deoxygalactonojirimycin) is an investigational pharmacological chaperone for the treatment of α-galactosidase A (α-Gal A) deficiency, which leads to Fabry disease, an X-linked, lysosomal storage disorder. The currently approved, biologicsbased therapy for Fabry disease is enzyme replacement therapy (ERT) with either agalsidase alfa (Replagal) or agalsidase beta (Fabrazyme). Based on preclinical data, migalastat HCl in combination with agalsidase is expected to result in the pharmacokinetic (PK) enhancement of agalsidase in plasma by increasing the systemic exposure of active agalsidase, thereby leading to increased cellular levels in disease-relevant tissues. This P..
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Funding Acknowledgements
This clinical trial, AT1001-013, its design, execution, data analysis, report writing, and manuscript preparation was funded by Amicus Therapeutics and GSK. Several authors left Amicus subsequent to the preparation of this manuscript. JJF went to Arvinas Inc. BAW and DJL went to OrPhi Therapeutics. CB went to The Parkinson's Institute and Clinical Center. PB went to Cymabay Therapeutics. While these companies now provide support in the form of salaries for authors JJF, BAW, DJL, CB, and PB, they in no way provided any support for this clinical trial. Clinical Research Investigators OGA and MT are affiliated with private companies which, however, did not provide support for this clinical trial. The specific roles of these authors are articulated in the "author contributions" section.