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Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.

Authors :: Jones S
Coker M
López AG
Sniadecki J
Mayhew J
Hensman P
Jurecka A
Source :: Molecular genetics and metabolism reports [Mol Genet Metab Rep] 2021 May 29; Vol. 28, pp. 100774. Date of Electronic Publication: 2021 May 29 (Print Publication: 2021).
Publication Year :: 2021
Abstract: Vestronidase alfa is an enzyme replacement therapy for mucopolysaccharidosis VII (MPS VII). In this open-label, phase 1/2 study, three subjects with MPS VII received intravenous vestronidase alfa administered every other week (QOW) for 14 weeks (2 mg/kg), followed by 24-week forced-dose titration (1, 4, and 2 mg/kg QOW; 8 weeks each), 36-week continuation (2 mg/kg), and long-term extension (4 mg/kg). Vestronidase alfa was well tolerated and led to dose-responsive, sustained reductions in urinary glycosaminoglycan excretion.<br />Competing Interests: Simon Jones: clinical trial investigator for study funded by Ultragenyx Pharmaceutical Inc. Mahmut Coker: clinical trial investigator for study funded by Ultragenyx Pharmaceutical Inc. Antonio González-Meneses López: clinical trial investigator for study funded by Ultragenyx Pharmaceutical Inc. Served on advisory board for Sanofi and Ultragenyx. Travel expenses from Sanofi, Ultragenyx, Shire. Pauline Hensman: clinical trial investigator for study funded by Ultragenyx Pharmaceutical Inc. Jennifer Sniadecki, Jill Mayhew, and Agnieszka Jurecka: employees and shareholders of Ultragenyx Pharmaceutical Inc.<br /> (© 2021 Published by Elsevier Inc.)