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


Jones S., ÇOKER M. , Gonzalez-Meneses Lopez A., Sniadecki J., Mayhew J., Hensman P., ...More

MOLECULAR GENETICS AND METABOLISM REPORTS, vol.28, 2021 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 28
  • Publication Date: 2021
  • Doi Number: 10.1016/j.ymgmr.2021.100774
  • Title of Journal : MOLECULAR GENETICS AND METABOLISM REPORTS
  • Keywords: Vestronidase alfa, Recombinant human beta-glucuronidase, Enzyme replacement therapy, Mucopolysaccharidosis VII, MPS VII, Phase 1/2

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.